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Terence R Flotte MD

TitleProfessor
Endowed TitleCelia and Isaac Haidak Professor
InstitutionUMass Chan Medical School
DepartmentPediatrics
AddressUMass Chan Medical School
55 Lake Avenue North S1-340
Worcester MA 01655
Phone508-856-2107
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    Other Positions
    InstitutionT.H. Chan School of Medicine
    DepartmentMicrobiology and Physiological Systems

    InstitutionT.H. Chan School of Medicine
    DepartmentPediatrics
    DivisionPediatric Pulmonology

    InstitutionT.H. Chan School of Medicine
    DepartmentRNA Therapeutics Institute

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentBiochemistry and Molecular Pharmacology

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentClinical Population Health Research

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentImmunology and Microbiology Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentInterdisciplinary Graduate Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentMasters in Clinical Investigation

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentMillennium MD/PhD Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentTranslational Science


    Collapse Biography 
    Collapse education and training
    University of New Orleans, New Orleans, LA, United StatesBSBiological Sciences
    Louisiana State University Medical Center, New Orleans, LA, United StatesMD

    Collapse Overview 
    Collapse overview

    Dr Flotte received his undergraduate degree in the biological sciences from the University of New Orleans in 1982, and his medical degree from the Louisiana State University School of Medicine in 1986. After serving his residency in pediatrics at Johns Hopkins University, he completed a pediatric pulmonary fellowshipa and postdoctoral training in molecular virology there in 1992.

    In 1995, Dr. Flotte and his colleagues at Johns Hopkins became the first to use adeno-associated virus, or AAV, as a vehicle to deliver corrective genes to targeted sites in the body, including the damaged airways of adults with cystic fibrosis.

    In 1996, Dr Flotte joined the faculty of the University of Florida and was appointed Associate Director of UF’s Powell Gene Therapy Center. In 2000, he was named Director of the Powell Center and founding Director of the newly established UF Genetics Institute, a cross-campus multidisciplinary unit encompassing gene therapy, human genetics, agricultural genetics and comparative genomics. In 2002, Flotte stepped down from these roles to accept the position of Chair of the Department of Pediatrics.

    An internationally known pioneer in human gene therapy, Flotte is currently investigating the use of gene therapy for genetic diseases that affect children, including cystic fibrosis, alpha-1 antitrypsin (AAT) deficiency, type I diabetes, and disorders of fatty acid oxidation. He is currently conducting Phase I trials with rAAV expressing alpha-1antitrypsin in AAT-deficient patients. He has also focused on the study of adeno-associated virus (AAV) vectors. Dr. Flotte's laboratory has also focused on the mechanisms of AAV persistence, since these represent the basis for a more profound understanding of the potential for long-term safe and effective gene therapy.

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    Research

    Recombinant adeno-associated virus (AAV) gene therapy vectors for, cystic fibrosis (CF), alpha-1-antitrypsin deficiency (AAT) and fatty oxidation disorders (FAO)

    adeno virusAAV is a non-pathogenic human parvovirus which is commonly isolated from the respiratory tract of humans (Figure 1A). AAV’s life cycle includes a unique mechanism for persistence in human cells by means of site-specific integration into a region of chromosome 19, the AAVS1 site (Figure 1B). In 1993, our group published the results of the first successful in vivo gene transfer with AAV. In that study, AAV vectors carrying the human CF transmembrane conductance regulator (CFTR) gene were delivered to the bronchial epithelium of rabbits. Efficient gene transfer was observed which persisted for over 6 months without any detectable toxicity. Subsequently, we studied AAV-CFTR gene transfer in rhesus macaques. Our studies in rhesus served two purposes: 1.We performed the primary toxicology study to justify beginning our phase I trial of AAV-CFTR administration in humans, and 2. we studied vector integration and persistence in vivo for the first time. Since then our laboratory has gone on to perform phase I clinical trials delivering AAV2 expressing the CFTR gene in CF patients. Much has been learned form the early trials including that paucity of AAV2 receptors on the luminal side of the airways and the weak promoter activity in early vectors. This limited the efficiency and efficacy of the viral vectors used in the early trials. To overcome this, the lab has designed a more robust expression cassette delivering a CFTR ‘mini’ gene and has determined that AAV1 is far superior to AAV2 at transducing airways. These new generation viral vectors for CF gene therapy are currently undergoing pre-clinical testing and will soon be tested in patients. Our laboratory has also pioneered phase I clinical studies using intra-muscular administration of rAAV2 as well as this more novel AAV1 serotype expressing the gene for alpha-1-antitrypsin (AAT) in AAT deficient patients. We are also developing hybrid rAAV-RNAi approaches to treat AAT deficient liver disease in which a toxic gain of function of the mutant protein contributes to the pathology. The laboratory is also involved in gene therapy for fatty oxidation disorders. Specifically we are developing gene delivery for the Acyl-CoA Dehydrogenases family enzymes that initiate the first step of the B-oxidation pathway.

    Current Studies in Cystic Fibrosis

    CFTR+/+Cystic fibrosis (CF), the most common lethal, single-gene disorder affecting Northern Europeans and North Americans, is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR is a chloride channel and a regulator of other ion channels, and many aspects of the CF phenotype are directly related to ion channel abnormalities attributable to CFTR mutation. What remains less clear, however, is how CFTR mutation leads to persistent endobronchial infection with Pseudomonas aeruginosa and severe airway inflammation which are the hallmarks of the lung pathology in CF. One theory, which remains controversial, contends that CFTR mutation primes CF cells to release greater quantities of pro-inflammatory cytokines than non-CF cells. One line of evidence in support of this theory is a Pseudomonas agarose bead airway infection model, in which CFTR knockout mice demonstrate greater weight loss and mortality and higher levels of pro-inflammatory cytokines than control mice. One of the limitations of this model has been its technical complexity and a wide variability of responses among individual animals. Because of the need for a technically simpler mouse model for inflammatory lung disease in CF, our laboratory has recently developed a new approach based on Aspergillus fumigatus (Af) sensitization and challenge. This model reproduces certain key immunologic and pathologic aspects of allergic bronchopulmonary aspergillosis (ABPA) an inflammatory lung phenotype that is much more common in CF patients than in any other clinical context. In the animal model studies, CFTR knockout mice demonstrated up-regulated levels of IL4 prior to sensitization, and subsequent to sensitization developed a hyper-IgE response and markedly divergent cytokine expression (including increased IL13, IL4, IL2, IL10, and KC) as compared with non-CF. While this CFTR-dependent inflammatory phenotype is potentially very promising, it has raised more questions than it answered. In particular, the divergence of expression of cytokines that are predominantly produced within lymphocytes, macrophages and other non-epithelial cells raises the possibility of an important role of CFTR itself in non-epithelial cells. Therefore our lab is currently studying which cell types contribute primarily to the CF-dependent inflammatory lung disease phenotype in Aspergillus-sensitized CF mice? What pathways or other mechanisms might be aberrantly regulated in affected cells?

    Gene and RNAi Therapy for Alpha-1 Antitrypsin

    Protease - Induced Lung DamageAlpha-1 antitrypsin (AAT) is one of the primary circulating serum antiproteases in humans. It inhibits a variety of serine proteinases, with neutrophil elastase being one of the most physiologically important, as well as inhibiting a number of metallo-proteinases and other pro-inflammatory and pro-apoptotic molecules. AAT is normally produced within hepatocytes and macrophages, where hepatocyte-derived AAT forms the bulk of the physiologic reserve of AAT. Approximately 4% of the North American and Northern European populations possess at least one copy of a mutant allele, known as PI*Z, which results from a single amino acid substitution of Lys for Glu at position 342. In the homozygous state, this mutation leads to severe deficiency of AAT, and can result in two distinct pathologic states: a lung disease which is primarily due to the loss of antiprotease function, and a liver disease (present to a significant degree in only 10% of patients) which is due to a toxic gain of function of the Z-AAT mutant protein. The mutation of Z-AAT leads to the loss of a crucial salt-bridge in a beta sheet region of the protein, allowing for the insertion of the reactive loop of a neighboring AAT molecule in between the two beta sheets. The structure of the resultant very stable loop-sheet polymers has been solved and provides insight into how the mutant may accumulate in a misfolded configuration. This accumulation within hepatocytes consistently results in a state of serum deficiency due to inefficient secretion. In individuals affected by AAT liver disease, it also triggers a cascade of aberrant signals within the hepatocyte, most likely the result of an unfolded protein response. However, the downstream details remain unclear, as does the reason for the disparity between those Z-homozygotes who develop liver disease and those who do not. Regardless of the precise mechanism by which Z-AAT conformational changes lead to liver disease, there is general consensus that molecular therapies for AAT liver disease should act by down-regulating Z-AAT. Our group has developed two different investigational clinical gene therapy products for gene augmentation of AAT as a potential therapy for the lung disease (rAAV2-AAT and rAAV1-AAT) (1, 4), and we have a third vector in development that is much more efficient for delivery of wild-type (M) AAT to hepatocytes (rAAV8-AAT). Given the need for down-regulation of Z-AAT, we have also begun to examine a number of innovative approaches to long-term expression of therapeutic RNAs using the recombinant adeno-associated virus (rAAV) platform, including spliceosome-mediated RNA trans-splicing (SMaRT), and rAAV-mediated in vivo delivery of RNAi in Z-AAT transgenic mice, a project which was recently published from our laboratory. The constructs in that paper utilized a pol III promoter (U6) to express short hairpin RNAs (shRNAs) targeting three different sites on the AAT molecule, but were not specific to the mutant allele. While it is feasible to direct silencing agents to the liver to decrease Z-AAT expression, while directing gene augmentation to other sites, the hepatocyte is most likely the optimal target for augmentation as well. Our lab is currently focusing on systematically developing RNAi-based approaches to Z-AAT down-regulation within hepatocytes and to devise appropriate strategies that might allow this to be combined with M-AAT gene augmentation

    Gene Therapy for Fatty Oxidation Disorders

    Genetic disorders of mitochondrial fatty acid oxidation (FAO) represent a relatively common class of metabolic disorders, exceeding more than 1 in 15,000 newborns. These disorders are associated with genes involved in the mitochondrial b-oxidation of fatty acids, which provide fuel once glycogen stores are depleted during prolonged fasting or during times of increased energy demands and physiological stress. During these circumstances liberation from triglycleride stores in adipose tissue causes free fatty acids circulate to the liver and striated muscle. After the fatty acids diffuse (short chain) or are actively transported (long chain) across the cellular and mitochondrial membranes, the oxidation of fatty acids occurs within the mitochondrial matrix. b-oxidation proceeds in a cyclical fashion resulting in the removal of sequential 2-carbon units as acetyl-CoA, for entry into the TCA cycle

    Importantly, the first step of each cycle in the mitochondrial matrix is catalyzed by an acyl-CoA dehydrogenase. This rate limiting function is performed by a family of enzymes that differ in their substrate specificity based on the carbon chain length of the acyl CoA molecule. The acyl-CoA dehydrogenases (ACDs) are a family of 5 mitochondrial enzymes involved in fatty acid and amino acid metabolism that catalyze the transfer of electrons from various acyl-CoA esters to electron transfer flavoprotein. Very long, medium and short chain acyl-CoA dehydrogenases (VLCAD, MCAD and SCAD) catalyze the first step in the b-oxidation cycle with substrate specificities of 16-, 8- and 4- carbon chains, respectively.

    cytosolCurrently our lab is applying rAAV-based transduction of skeletal muscle for those FAO deficiencies that are most common in humans, medium chain acyl CoA dehydrogenase (MCAD) and very long chain acyl CoA dehydrodgenase (VLCAD). Over the past 5 years, mouse models have become available for each of these disorders, and mass newborn screening for FAO disorders has been initiated in many states. Our prior work has relied heavily on rAAV1 pseudotyped vectors for skeletal muscle transduction. The emergence of rAAV9-based vectors now provides the potential for even greater efficiency in using transduction of skeletal muscle and cardiac muscle as a platform for gene therapy of systemic genetic diseases. We are investigating to compare rAAV1 and rAAV9 in the context of a translational application to VLCAD and MCAD deficiencies. Specifically, the feasibility of molecular and biochemical correction of VLCAD and MCAD deficiencies is tested in mouse models of these deficiencies. Our primary focus is on determining whether rAAV9 (vs. rAAV1)-mediated transduction of skeletal and cardiac muscle will result in greater improvement of acyl carnitine profiles (i.e., clearance of abnormally high levels of fatty acyl carnitine metabolites that are characteristic of this disorder) and MRS profiles. We are also going to develop formal preclinical toxicology studies of muscle delivery of rAAV9 or rAAV1-VLCAD and rAAV9 or rAAV1-MCAD which will be completed in anticipation of new phase I clinical trials of gene therapy for these FAO disorders.



    Collapse Bibliographic 
    Collapse selected publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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    1. Ke H, Guay KP, Flotte TR, Gierasch LM, Gershenson A, Hebert DN. Secretion of functional ?1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy. Proc Natl Acad Sci U S A. 2022 08 02; 119(31):e2206103119. PMID: 35901208.
      Citations:    Fields:    
    2. Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M. Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther. 2022 07 06; 30(7):2416-2428. PMID: 35585789.
      Citations:    Fields:    Translation:Humans
    3. Gruntman AM, Flotte TR. Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient. Hum Gene Ther. 2022 05; 33(9-10):477-478. PMID: 35576406.
      Citations:    Fields:    Translation:Animals
    4. Zieger M, Borel F, Greer C, Gernoux G, Blackwood M, Flotte TR, Mueller C. Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in ?1-antitrypsin null mice. Mol Ther Methods Clin Dev. 2022 Jun 09; 25:425-438. PMID: 35592360.
      Citations:    
    5. Flotte TR, Gessler DJ. Gene Therapy for Rare Neurological Disorders. Clin Pharmacol Ther. 2022 04; 111(4):743-757. PMID: 35102556.
      Citations:    Fields:    Translation:Humans
    6. Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH, Martin DR, Gray-Edwards HL, Sena-Esteves M. AAV gene therapy for Tay-Sachs disease. Nat Med. 2022 02; 28(2):251-259. PMID: 35145305.
      Citations: 3     Fields:    Translation:HumansCells
    7. Flotte TR. Precision of Exon Skipping with U7 Constructs. Hum Gene Ther. 2021 11; 32(21-22):1315-1316. PMID: 34780289.
      Citations:    Fields:    Translation:Humans
    8. Arjomandnejad M, Sylvia K, Blackwood M, Nixon T, Tang Q, Muhuri M, Gruntman AM, Gao G, Flotte TR, Keeler AM. Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells. Mol Ther Methods Clin Dev. 2021 Dec 10; 23:490-506. PMID: 34853797.
      Citations:    
    9. Flotte TR. ESGCT 2021: Virtually Pan-European. Hum Gene Ther. 2021 Oct; 32(19-20):978. PMID: 34662234.
      Citations:    Fields:    
    10. Batista AR, Flotte TR. In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis. Mol Ther. 2021 09 01; 29(9):2633-2634. PMID: 34433081.
      Citations:    Fields:    Translation:Humans
    11. Flotte TR. Base Editing to the Rescue. Hum Gene Ther. 2021 Jul; 32(13-14):647-648. PMID: 34283641.
      Citations:    Fields:    
    12. Flotte TR, Gao G. Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword. Hum Gene Ther. 2021 07; 32(13-14):645-646. PMID: 34283643.
      Citations:    Fields:    Translation:HumansCells
    13. Flotte TR, Gao G. It Is Time to Call Anti-Asian Bias What It Is: Racism. Hum Gene Ther. 2021 May; 32(9-10):431-432. PMID: 34003044.
      Citations:    Fields:    Translation:Humans
    14. Flotte TR, Larkin AC, Fischer MA, Chimienti SN, DeMarco DM, Fan PY, Collins MF. In Reply to Ramotshwana et al. Acad Med. 2021 05 01; 96(5):e15-e16. PMID: 33570854.
      Citations:    Fields:    Translation:HumansCellsPHPublic Health
    15. Flotte TR. Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia. Hum Gene Ther. 2021 May; 32(9-10):449-450. PMID: 34003043.
      Citations:    Fields:    Translation:Humans
    16. Flotte TR, Lord BT, Siedman J. Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives. Pediatrics. 2021 05; 147(5). PMID: 33863844.
      Citations:    Fields:    Translation:Humans
    17. Liu P, Liang SQ, Zheng C, Mintzer E, Zhao YG, Ponnienselvan K, Mir A, Sontheimer EJ, Gao G, Flotte TR, Wolfe SA, Xue W. Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. Nat Commun. 2021 04 09; 12(1):2121. PMID: 33837189.
      Citations: 39     Fields:    Translation:HumansAnimalsCells
    18. Adair JE, Rubinger KS, Flotte TR. Women Lead Gene Therapy Science in 2021. Hum Gene Ther. 2021 04; 32(7-8):317-318. PMID: 33891503.
      Citations:    Fields:    Translation:Humans
    19. Qin W, Xu G, Tai PWL, Wang C, Luo L, Li C, Hu X, Xue J, Lu Y, Zhou Q, Wei Q, Wen T, Hu J, Xiao Y, Yang L, Li W, Flotte TR, Wei Y, Gao G. Large-scale molecular epidemiological analysis of AAV in a cancer patient population. Oncogene. 2021 04; 40(17):3060-3071. PMID: 33782545.
      Citations: 2     Fields:    Translation:HumansCells
    20. Jacobson CE, Beeler WH, Griffith KA, Flotte TR, Byington CL, Jagsi R. Common pathways to Dean of Medicine at U.S. medical schools. PLoS One. 2021; 16(3):e0249078. PMID: 33765033.
      Citations:    Fields:    Translation:Humans
    21. Dasgupta I, Flotte TR, Keeler AM. CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing. Hum Gene Ther. 2021 03; 32(5-6):275-293. PMID: 33750221.
      Citations: 6     Fields:    Translation:Humans
    22. Flotte TR. CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing. Hum Gene Ther. 2021 03; 32(5-6):235-236. PMID: 33750218.
      Citations: 1     Fields:    Translation:Cells
    23. Flotte TR. Liver targeting with rAAV7: balancing tropism with immune profiles. Gene Ther. 2021 04; 28(3-4):115-116. PMID: 33547423.
      Citations:    Fields:    Translation:Cells
    24. Büning H, Baker AH, Griesenbach U, Flotte TR, Thrasher AJ. Gene and Cell Therapy for Inherited and Acquired Immune Deficiency. Hum Gene Ther. 2021 01; 32(1-2):1-3. PMID: 33471605.
      Citations:    Fields:    Translation:Humans
    25. Flotte TR. Science Policy and Funding Implications of the 2020 U.S. Election. Hum Gene Ther. 2020 12; 31(23-24):1215-1216. PMID: 33337269.
      Citations:    Fields:    Translation:Humans
    26. Flotte TR. Writing the Story of Gene Editing and CRISPR: An Interview with Kevin Davies, PhD. Hum Gene Ther. 2020 12; 31(23-24):1217-1220. PMID: 33337270.
      Citations:    Fields:    Translation:Humans
    27. Tang Q, Keeler AM, Zhang S, Su Q, Lyu Z, Cheng Y, Gao G, Flotte TR. Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus. Biores Open Access. 2020; 9(1):219-228. PMID: 33117614.
      Citations:    
    28. Flotte TR, Larkin AC, Fischer MA, Chimienti SN, DeMarco DM, Fan PY, Collins MF. Accelerated Graduation and the Deployment of New Physicians During the COVID-19 Pandemic. Acad Med. 2020 10; 95(10):1492-1494. PMID: 32520751.
      Citations: 10     Fields:    Translation:HumansCellsPHPublic Health
    29. Wilson JM, Flotte TR. Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy. Hum Gene Ther. 2020 07; 31(13-14):695-696. PMID: 32605399.
      Citations: 50     Fields:    Translation:Humans
    30. Flotte TR, Adair JE. Black Lives Matter to Gene Therapy. Hum Gene Ther. 2020 07; 31(13-14):700. PMID: 32716736.
      Citations:    Fields:    Translation:Humans
    31. Jagsi R, Means O, Lautenberger D, Jones RD, Griffith KA, Flotte TR, Gordon LK, Rexrode KM, Wagner LW, Chatterjee A. Women's Representation Among Members and Leaders of National Medical Specialty Societies. Acad Med. 2020 07; 95(7):1043-1049. PMID: 31625994.
      Citations: 4     Fields:    Translation:Humans
    32. Taghian T, Horn E, Shazeeb MS, Bierfeldt LJ, Tuominen SM, Koehler J, Fernau D, Bertrand S, Frey S, Cataltepe OI, Gounis MJ, Abayazeed AH, Flotte TR, Sena-Esteves M, Gray-Edwards HL. Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model. Hum Gene Ther. 2020 06; 31(11-12):617-625. PMID: 32363942.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    33. Flotte TR. Barrie J. Carter: The Science and the Scientist. Hum Gene Ther. 2020 05; 31(9-10):489-490. PMID: 32384022.
      Citations:    Fields:    Translation:HumansCells
    34. Flotte TR. Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors. Hum Gene Ther. 2020 04; 31(7-8):398-399. PMID: 32302233.
      Citations: 10     Fields:    Translation:HumansCells
    35. Flotte TR. A Message to Our Community in the Midst of the COVID-19 Pandemic. Hum Gene Ther. 2020 04; 31(7-8):397. PMID: 32212981.
      Citations:    Fields:    
    36. Flotte TR. Impact on Women's Health: Gene Therapy in Gynecologic Oncology. Hum Gene Ther. 2020 03; 31(5-6):271-272. PMID: 32202458.
      Citations:    Fields:    Translation:Humans
    37. Flotte TR. Free Gene and Cell Therapy Services Offered by the National Heart, Lung, and Blood Institute of the National Institutes of Health. Hum Gene Ther. 2020 02; 31(3-4):133-134. PMID: 32073927.
      Citations:    Fields:    
    38. Gernoux G, Gruntman AM, Blackwood M, Zieger M, Flotte TR, Mueller C. Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans. Mol Ther. 2020 03 04; 28(3):747-757. PMID: 31982038.
      Citations: 9     Fields:    Translation:HumansAnimalsCells
    39. Tang Q, Gernoux G, Cheng Y, Flotte T, Mueller C. Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model. Methods Mol Biol. 2020; 2164:75-85. PMID: 32607885.
      Citations:    Fields:    Translation:HumansAnimalsCells
    40. Chimienti SN, DeMarco DM, Flotte TR, Collins MF. In Reply to Signer and Curtin and to Goyal et al. Acad Med. 2020 01; 95(1):9. PMID: 31860618.
      Citations:    Fields:    
    41. Flotte TR, Gao G. Ringing in the Changes. Hum Gene Ther. 2020 01; 31(1-2):4. PMID: 31967918.
      Citations:    Fields:    
    42. Flotte TR, Gao G. 2020: Gene Therapy Enters Its Fourth Decade. Hum Gene Ther. 2020 01; 31(1-2):2-3. PMID: 31967921.
      Citations: 2     Fields:    
    43. Flotte TR. Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy. Hum Gene Ther. 2020 01; 31(1-2):12-13. PMID: 31967920.
      Citations:    Fields:    Translation:Humans
    44. Flotte TR, Gao G. Prime Editing: A Novel Cas9-Reverse Transcriptase Fusion May Revolutionize Genome Editing. Hum Gene Ther. 2019 12; 30(12):1445-1446. PMID: 31860398.
      Citations: 2     Fields:    Translation:HumansCells
    45. Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, Maguire AS, Martin DR, Fernau D, Batista AR, Kuchel T, Christou C, Perumal R, Chandra S, Gamlin PD, Bertrand SG, Flotte TR, McKenna-Yasek D, Tai PWL, Aronin N, Gounis MJ, Sena-Esteves M, Gray-Edwards HL. A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. Mol Ther. 2020 02 05; 28(2):411-421. PMID: 31813800.
      Citations: 22     Fields:    Translation:HumansAnimalsCells
    46. Flotte TR. Epigenome Editing Strategies for Low Back Pain. Hum Gene Ther. 2019 09; 30(9):1037-1038. PMID: 31573390.
      Citations:    Fields:    Translation:Humans
    47. Flotte TR. Getting Tough on Capsid Screening: Tough Decoys Enable Barcoding of Vectors Capable of both Entry and Expression. Hum Gene Ther. 2019 08; 30(8):919-920. PMID: 31418605.
      Citations:    Fields:    Translation:HumansAnimalsCells
    48. Keeler AM, Flotte TR. Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? Annu Rev Virol. 2019 09 29; 6(1):601-621. PMID: 31283441.
      Citations: 76     Fields:    Translation:HumansAnimalsCells
    49. Flotte TR. Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing. Hum Gene Ther. 2019 07; 30(7):793. PMID: 31334692.
      Citations:    Fields:    Translation:HumansAnimals
    50. Zieger M, Keeler AM, Flotte TR, ElMallah MK. AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency. J Inherit Metab Dis. 2019 09; 42(5):870-877. PMID: 30993714.
      Citations: 3     Fields:    Translation:AnimalsCells
    51. Barzel A, Flotte TR. New Horizons for Immune Gene Therapy. Hum Gene Ther. 2019 04; 30(4):379-380. PMID: 30950655.
      Citations:    Fields:    Translation:Humans
    52. Adair JE, Flotte TR. What the Gene Therapy Community Should Do About Sexual Harassment. Hum Gene Ther. 2019 03; 30(3):249-251. PMID: 30755038.
      Citations:    Fields:    
    53. Chimienti SN, DeMarco DM, Flotte TR, Collins MF. Assuring Integrity in the Residency Match Process. Acad Med. 2019 03; 94(3):321-323. PMID: 30431456.
      Citations: 2     Fields:    Translation:Humans
    54. Gruntman AM, Gernoux G, Tang Q, Ye GJ, Knop DR, Wang G, Benson J, Coleman KE, Keeler AM, Mueller C, Chicoine LG, Chulay JD, Flotte TR. Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study. Mol Ther Methods Clin Dev. 2019 Jun 14; 13:233-242. PMID: 30828586.
      Citations:    
    55. Flotte TR. Top Five Gene Therapy Stories of 2019. Hum Gene Ther. 2019 01; 30(1):1-2. PMID: 30628861.
      Citations:    Fields:    Translation:Humans
    56. Flotte TR. The Year in Review: The Top Five Papers of 2018. Hum Gene Ther. 2018 12; 29(12):1339-1340. PMID: 30757924.
      Citations:    Fields:    Translation:HumansAnimalsCells
    57. Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G. Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer. Mol Ther Methods Clin Dev. 2018 Dec 14; 11:65-72. PMID: 30397628.
      Citations:    
    58. Flotte TR, Lu S. DNA Vaccination in 2018: An Update. Hum Gene Ther. 2018 09; 29(9):963-965. PMID: 30230936.
      Citations: 1     Fields:    Translation:HumansPHPublic Health
    59. Flotte TR. Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. Hum Gene Ther. 2018 08; 29(8):843-844. PMID: 30096037.
      Citations: 1     Fields:    Translation:Humans
    60. Pennesi ME, Weleber RG, Yang P, Whitebirch C, Thean B, Flotte TR, Humphries M, Chegarnov E, Beasley KN, Stout JT, Chulay JD. Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. Hum Gene Ther. 2018 12; 29(12):1428-1437. PMID: 29869534.
      Citations: 14     Fields:    Translation:HumansCellsCTClinical Trials
    61. Flotte TR. Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy. Hum Gene Ther. 2018 06; 29(6):641-642. PMID: 29902084.
      Citations: 1     Fields:    Translation:HumansAnimals
    62. Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther. 2018 08; 29(8):853-860. PMID: 29597895.
      Citations: 23     Fields:    Translation:HumansAnimalsCells
    63. Gruntman AM, Flotte TR. The rapidly evolving state of gene therapy. FASEB J. 2018 04; 32(4):1733-1740. PMID: 31282760.
      Citations: 11     Fields:    Translation:HumansCells
    64. Flotte TR. Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach. Hum Gene Ther. 2018 04; 29(4):401-402. PMID: 29668376.
      Citations:    Fields:    Translation:HumansCellsPHPublic Health
    65. Wang D, Li S, Gessler DJ, Xie J, Zhong L, Li J, Tran K, Van Vliet K, Ren L, Su Q, He R, Goetzmann JE, Flotte TR, Agbandje-McKenna M, Gao G. A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates. Mol Ther Methods Clin Dev. 2018 Jun 15; 9:234-246. PMID: 29766031.
      Citations:    
    66. Borel F, Sun H, Zieger M, Cox A, Cardozo B, Li W, Oliveira G, Davis A, Gruntman A, Flotte TR, Brodsky MH, Hoffman AM, Elmallah MK, Mueller C. Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema. Proc Natl Acad Sci U S A. 2018 03 13; 115(11):2788-2793. PMID: 29453277.
      Citations: 29     Fields:    Translation:HumansAnimals
    67. Flotte TR, Büning H. Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First. Hum Gene Ther. 2018 03; 29(3):283-284. PMID: 29378415.
      Citations: 14     Fields:    Translation:AnimalsCells
    68. Flotte TR, Gao ??? G. Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century ? 21. Hum Gene Ther. 2018 02; 29(2):97. PMID: 29446999.
      Citations:    Fields:    Translation:Humans
    69. Bates CK, Jagsi R, Gordon LK, Travis E, Chatterjee A, Gillis M, Means O, Chaudron L, Ganetzky R, Gulati M, Fivush B, Sharma P, Grover A, Lautenberger D, Flotte TR. It Is Time for Zero Tolerance for Sexual Harassment in Academic Medicine. Acad Med. 2018 02; 93(2):163-165. PMID: 29116986.
      Citations: 24     Fields:    Translation:Humans
    70. Flotte TR. Gene and Cell Therapy in 2018: A Look Ahead. Hum Gene Ther. 2018 01; 29(1):1. PMID: 29328843.
      Citations:    Fields:    
    71. Flotte TR. Therapeutic Advances in Germany and Beyond. Hum Gene Ther. 2017 12; 28(12):1117. PMID: 29265969.
      Citations:    Fields:    Translation:Humans
    72. Flotte TR, Daniels E, Benson J, Bevett-Rose JM, Cornetta K, Diggins M, Johnston J, Sepelak S, van der Loo JCM, Wilson JM, McDonald CL. The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute. Hum Gene Ther Clin Dev. 2017 12; 28(4):178-186. PMID: 29130351.
      Citations:    Fields:    Translation:HumansCells
    73. Flotte TR. European Society of Gene and Cell Therapy (ESGCT) at 25: A Gene Therapy Community at Its Prime and on the Move. Hum Gene Ther. 2017 11; 28(11):940. PMID: 29035113.
      Citations:    Fields:    Translation:Humans
    74. Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of ?-1 Antitrypsin Deficiency. Mol Ther. 2017 11 01; 25(11):2477-2489. PMID: 29032169.
      Citations: 30     Fields:    Translation:HumansAnimalsCells
    75. Flotte TR. Gene Therapy for Alcoholism and Other Substance Use Disorders. Hum Gene Ther. 2017 09; 28(9):701-702. PMID: 28854046.
      Citations:    Fields:    Translation:Humans
    76. Flotte TR. Adeno-Associated Virus-Human Bocavirus 1 Chimeric Vectors: Ferreting Out Their Role in Airway Gene Therapy. Hum Gene Ther. 2017 08; 28(8):611. PMID: 28796554.
      Citations:    Fields:    Translation:HumansAnimalsCellsPHPublic Health
    77. Flotte TR. Sleeping Beauty Awakens New Interest. Hum Gene Ther. 2017 07; 28(7):533. PMID: 28719250.
      Citations:    Fields:    Translation:Cells
    78. Flotte TR. One More Controversy: Adeno-Associated Virus in Stem Cells. Hum Gene Ther. 2017 06; 28(6):449. PMID: 28604173.
      Citations:    Fields:    Translation:Cells
    79. Bharel M, Antman K, Berman H, Dimitri D, Flier J, Flotte T. In Reply to Manion and Khan. Acad Med. 2017 06; 92(6):725-726. PMID: 28557909.
      Citations:    Fields:    
    80. Keeler AM, ElMallah MK, Flotte TR. Gene Therapy 2017: Progress and Future Directions. Clin Transl Sci. 2017 07; 10(4):242-248. PMID: 28383804.
      Citations: 46     Fields:    Translation:HumansCells
    81. Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR. 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency. Mol Ther. 2017 06 07; 25(6):1387-1394. PMID: 28408179.
      Citations: 43     Fields:    Translation:HumansCells
    82. Flotte TR, Gao G. AAV Is Now a Medicine: We Had Better Get This Right. Hum Gene Ther. 2017 04; 28(4):307. PMID: 28414552.
      Citations:    Fields:    Translation:HumansCells
    83. Wilkins O, Keeler AM, Flotte TR. CAR T-Cell Therapy: Progress and Prospects. Hum Gene Ther Methods. 2017 04; 28(2):61-66. PMID: 28330372.
      Citations: 16     Fields:    Translation:HumansCells
    84. Gruntman AM, Su L, Flotte TR. Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups. Hum Gene Ther. 2017 03; 28(3):228-230. PMID: 28319444.
      Citations: 5     Fields:    Translation:AnimalsCells
    85. Flotte TR. The Negative Effects of Immigration Restrictions on the Gene Therapy Community. Hum Gene Ther. 2017 03; 28(3):227. PMID: 28319443.
      Citations:    Fields:    Translation:Humans
    86. Stoica L, Keeler AM, Xiong L, Kalfopoulos M, Desrochers K, Brown RH, Sena-Esteves M, Flotte TR, ElMallah MK. Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model. Am J Respir Cell Mol Biol. 2017 03; 56(3):405-408. PMID: 28248134.
      Citations: 7     Fields:    Translation:HumansAnimals
    87. Flotte TR. Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy? Hum Gene Ther. 2017 02; 28(2):147-148. PMID: 28186848.
      Citations: 4     Fields:    Translation:HumansCells
    88. Calcedo R, Somanathan S, Qin Q, Betts MR, Rech AJ, Vonderheide RH, Mueller C, Flotte TR, Wilson JM. Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for ?-1-antitrypsin deficiency. Proc Natl Acad Sci U S A. 2017 02 14; 114(7):1655-1659. PMID: 28137880.
      Citations: 25     Fields:    Translation:HumansCellsCTClinical Trials
    89. Tang Q, Gruntman AM, Flotte TR. Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA. Methods Mol Biol. 2017; 1639:211-216. PMID: 28752460.
      Citations: 1     Fields:    Translation:Humans
    90. Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275. PMID: 28752467.
      Citations: 3     Fields:    Translation:Humans
    91. Flotte TR. The Science Policy Implications of a Trump Presidency. Hum Gene Ther. 2017 01; 28(1):1-2. PMID: 27922749.
      Citations: 2     Fields:    Translation:Humans
    92. Antman KH, Berman HA, Flotte TR, Flier J, Dimitri DM, Bharel M. Developing Core Competencies for the Prevention and Management of Prescription Drug Misuse: A Medical Education Collaboration in Massachusetts. Acad Med. 2016 Oct; 91(10):1348-1351. PMID: 27532868.
      Citations: 15     Fields:    
    93. Naldini L, Parker GC, Flotte TR. The Renaissance of Gene and Cell Therapy: Florence 2016. Hum Gene Ther. 2016 10; 27(10):727-728. PMID: 29112477.
      Citations:    Fields:    Translation:Humans
    94. Flotte TR. Gene Drives: Biological Shield or Ecological Menace? Hum Gene Ther. 2016 08; 27(8):561-2. PMID: 27513596.
      Citations:    Fields:    Translation:Humans
    95. Flotte TR. The Target's the Thing. Hum Gene Ther. 2016 07; 27(7):477. PMID: 27428657.
      Citations:    Fields:    Translation:HumansCells
    96. Flotte TR. Why Human Gene Therapy Scientists Should Care About Model Organisms. Hum Gene Ther. 2016 May; 27(5):339-40. PMID: 27082033.
      Citations:    Fields:    Translation:HumansAnimals
    97. Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. Ophthalmology. 2016 07; 123(7):1606-20. PMID: 27102010.
      Citations: 69     Fields:    Translation:HumansCellsCTClinical Trials
    98. Flotte TR. A Gene Therapy Scientist's Life Well-Lived. Hum Gene Ther. 2016 Apr; 27(4):279. PMID: 27074150.
      Citations:    Fields:    Translation:Humans
    99. Loring HS, ElMallah MK, Flotte TR. Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans. Hum Gene Ther Methods. 2016 Apr; 27(2):49-58. PMID: 26895204.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    100. Flotte TR. Special Note from the Editor-in-Chief. Hum Gene Ther. 2016 Feb; 27(2):95. PMID: 26886829.
      Citations:    Fields:    Translation:Humans
    101. Agre P, Bertozzi C, Bissell M, Campbell KP, Cummings RD, Desai UR, Estes M, Flotte T, Fogleman G, Gage F, Ginsburg D, Gordon JI, Hart G, Hascall V, Kiessling L, Kornfeld S, Lowe J, Magnani J, Mahal LK, Medzhitov R, Roberts RJ, Sackstein R, Sarkar R, Schnaar R, Schwartz N, Varki A, Walt D, Weissman I. Training the next generation of biomedical investigators in glycosciences. J Clin Invest. 2016 Feb; 126(2):405-8. PMID: 26829621.
      Citations: 15     Fields:    Translation:HumansAnimals
    102. Schnepp BC, Chulay JD, Ye GJ, Flotte TR, Trapnell BC, Johnson PR. Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle. Hum Gene Ther. 2016 Jan; 27(1):32-42. PMID: 26650966.
      Citations: 10     Fields:    Translation:HumansCells
    103. Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A. Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? Hum Gene Ther. 2015 Dec; 26(12):779-81. PMID: 26690810.
      Citations: 31     Fields:    Translation:HumansCells
    104. Flotte TR. The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy. Hum Gene Ther. 2015 Dec; 26(12):782. PMID: 26690811.
      Citations: 1     Fields:    Translation:Humans
    105. Flotte TR. Transcription Activator-Like Nucleases Enable Allogeneic Chimeric Antigen Receptor-T Cell Therapy in Humans. Hum Gene Ther. 2015 Nov; 26(11):iv. PMID: 26565678.
      Citations:    Fields:    Translation:HumansCells
    106. Li S, Ling C, Zhong L, Li M, Su Q, He R, Tang Q, Greiner DL, Shultz LD, Brehm MA, Flotte TR, Mueller C, Srivastava A, Gao G. Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors. Mol Ther. 2015 Dec; 23(12):1867-76. PMID: 26403887.
      Citations: 42     Fields:    Translation:HumansAnimalsCells
    107. Gruntman AM, Flotte TR. Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods. Hum Gene Ther Clin Dev. 2015 Sep; 26(3):159-64. PMID: 26357010.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    108. Flotte TR. Ethical Implications of the Cost of Molecularly Targeted Therapies. Hum Gene Ther. 2015 Sep; 26(9):573-4. PMID: 26355414.
      Citations: 4     Fields:    Translation:Humans
    109. Flotte TR. The End of the Beginning of Gene Therapy. Hum Gene Ther. 2015 Jul; 26(7):407-8. PMID: 26176429.
      Citations:    Fields:    
    110. Gruntman AM, Flotte TR. Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency. Hum Gene Ther Methods. 2015 Jun; 26(3):77-81. PMID: 26067712.
      Citations: 4     Fields:    Translation:HumansAnimalsCells
    111. Flotte TR. Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question? Hum Gene Ther. 2015 May; 26(5):245-6. PMID: 25927157.
      Citations: 4     Fields:    Translation:HumansCells
    112. Gruntman AM, Su L, Su Q, Gao G, Mueller C, Flotte TR. Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials. Hum Gene Ther Methods. 2015 Apr; 26(2):71-6. PMID: 25819833.
      Citations: 11     Fields:    Translation:HumansAnimalsCells
    113. Loring HS, Flotte TR. Current status of gene therapy for ?-1 antitrypsin deficiency. Expert Opin Biol Ther. 2015 Mar; 15(3):329-36. PMID: 25363251.
      Citations: 11     Fields:    Translation:HumansAnimalsCells
    114. Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. PMID: 24981438.
      Citations: 7     Fields:    Translation:Humans
    115. Flotte TR, Mueller C. What is suppression of anti-adeno-associated virus capsid T-cells achieving? Hum Gene Ther. 2014 Mar; 25(3):178-9. PMID: 24628440.
      Citations: 2     Fields:    Translation:HumansCells
    116. Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG, Messina L, Tang Q, Rouhani FN, Campbell-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M, Calcedo R, Somanathan S, Richman LP, Vonderheide RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR. Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest. 2013 Dec; 123(12):5310-8. PMID: 24231351.
      Citations: 79     Fields:    Translation:HumansCellsCTClinical Trials
    117. Flotte TR. Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy. Mol Ther. 2013 Nov; 21(11):1976-81. PMID: 24201212.
      Citations: 9     Fields:    Translation:HumansCells
    118. Ahmed SS, Li H, Cao C, Sikoglu EM, Denninger AR, Su Q, Eaton S, Liso Navarro AA, Xie J, Szucs S, Zhang H, Moore C, Kirschner DA, Seyfried TN, Flotte TR, Matalon R, Gao G. A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice. Mol Ther. 2013 Dec; 21(12):2136-47. PMID: 23817205.
      Citations: 47     Fields:    Translation:HumansAnimalsCells
    119. Zhong L, Malani N, Li M, Brady T, Xie J, Bell P, Li S, Jones H, Wilson JM, Flotte TR, Bushman FD, Gao G. Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. Hum Gene Ther. 2013 May; 24(5):520-5. PMID: 23621841.
      Citations: 21     Fields:    Translation:Cells
    120. Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9. PMID: 23527792.
      Citations: 11     Fields:    Translation:HumansCells
    121. Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther. 2013 Feb; 21(2):266-8. PMID: 23369965.
      Citations: 7     Fields:    Translation:Humans
    122. Gruntman AM, Bish LT, Mueller C, Sweeney HL, Flotte TR, Gao G. Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:Unit 14D.3. PMID: 23408131.
      Citations: 11     Fields:    Translation:AnimalsCells
    123. Bouchecareilh M, Hutt DM, Szajner P, Flotte TR, Balch WE. Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of ?1-antitrypsin deficiency. J Biol Chem. 2012 Nov 02; 287(45):38265-78. PMID: 22995909.
      Citations: 31     Fields:    Translation:HumansCells
    124. Bickford JS, Mueller C, Newsom KJ, Barilovits SJ, Beachy DE, Herlihy JD, Keeler B, Flotte TR, Nick HS. Effect of allergy and inflammation on eicosanoid gene expression in CFTR deficiency. J Cyst Fibros. 2013 May; 12(3):258-65. PMID: 22985691.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    125. Gruntman AM, Mueller C, Flotte TR, Gao G. Gene transfer in the lung using recombinant adeno-associated virus. Curr Protoc Microbiol. 2012 Aug; Chapter 14:Unit14D.2. PMID: 22875566.
      Citations: 6     Fields:    Translation:AnimalsCells
    126. Keeler AM, Flotte TR. Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome. Hum Gene Ther. 2012 Jun; 23(6):548-56. PMID: 22642257.
      Citations: 9     Fields:    Translation:Humans
    127. Lockett AD, Van Demark M, Gu Y, Schweitzer KS, Sigua N, Kamocki K, Fijalkowska I, Garrison J, Fisher AJ, Serban K, Wise RA, Flotte TR, Mueller C, Presson RG, Petrache HI, Tuder RM, Petrache I. Effect of cigarette smoke exposure and structural modifications on the ?-1 Antitrypsin interaction with caspases. Mol Med. 2012 May 09; 18:445-54. PMID: 22245800.
      Citations: 24     Fields:    Translation:HumansAnimals
    128. Keeler AM, Conlon T, Walter G, Zeng H, Shaffer SA, Dungtao F, Erger K, Cossette T, Tang Q, Mueller C, Flotte TR. Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy. Mol Ther. 2012 Jun; 20(6):1131-8. PMID: 22395529.
      Citations: 10     Fields:    Translation:AnimalsCells
    129. Xie J, Ameres SL, Friedline R, Hung JH, Zhang Y, Xie Q, Zhong L, Su Q, He R, Li M, Li H, Mu X, Zhang H, Broderick JA, Kim JK, Weng Z, Flotte TR, Zamore PD, Gao G. Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Nat Methods. 2012 Mar 04; 9(4):403-9. PMID: 22388288.
      Citations: 118     Fields:    Translation:AnimalsCells
    130. Bickford JS, Newsom KJ, Herlihy JD, Mueller C, Keeler B, Qiu X, Walters JN, Su N, Wallet SM, Flotte TR, Nick HS. Induction of group IVC phospholipase A2 in allergic asthma: transcriptional regulation by TNF? in bronchoepithelial cells. Biochem J. 2012 Feb 15; 442(1):127-37. PMID: 22082005.
      Citations: 9     Fields:    Translation:HumansAnimalsCells
    131. Mueller C, Tang Q, Gruntman A, Blomenkamp K, Teckman J, Song L, Zamore PD, Flotte TR. Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. Mol Ther. 2012 03; 20(3):590-600. PMID: 22252449.
      Citations: 49     Fields:    Translation:HumansAnimalsCells
    132. Ettinger WH, Flotte TR. The role of gene and cell therapy in the era of health care reform. Hum Gene Ther. 2011 Nov; 22(11):1307-9. PMID: 22023350.
      Citations: 1     Fields:    Translation:Humans
    133. Marella M, Seo BB, Flotte TR, Matsuno-Yagi A, Yagi T. No immune responses by the expression of the yeast Ndi1 protein in rats. PLoS One. 2011; 6(10):e25910. PMID: 21991386.
      Citations: 12     Fields:    Translation:AnimalsCells
    134. Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, Rouhani F, Campbell-Thompson M, Yachnis AT, Sandhaus RA, McElvaney NG, Mueller C, Messina LM, Wilson JM, Brantly M, Knop DR, Ye GJ, Chulay JD. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing ?1-antitrypsin: interim results. Hum Gene Ther. 2011 Oct; 22(10):1239-47. PMID: 21609134.
      Citations: 151     Fields:    Translation:HumansCellsCTClinical Trials
    135. Flotte TR, Frederickson RM, Lowenstein PR, Mueller C. Moving forward toward a cure for Parkinson's: neuropathology of the nigrostriatal pathway determines the location of growth factor delivery. Mol Ther. 2011 May; 19(5):827-9. PMID: 21532611.
      Citations:    Fields:    Translation:AnimalsCells
    136. Flotte TR, Mueller C. Gene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011 Apr 15; 20(R1):R87-92. PMID: 21498872.
      Citations: 19     Fields:    Translation:HumansAnimalsCells
    137. Chen KA, Cruz PE, Lanuto DJ, Flotte TR, Borchelt DR, Srivastava A, Zhang J, Steindler DA, Zheng T. Cellular fusion for gene delivery to SCA1 affected Purkinje neurons. Mol Cell Neurosci. 2011 May; 47(1):61-70. PMID: 21420496.
      Citations: 16     Fields:    Translation:HumansAnimalsCells
    138. Silver JN, Elder M, Conlon T, Cruz P, Wright AJ, Srivastava A, Flotte TR. Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency. Hum Gene Ther. 2011 Aug; 22(8):935-49. PMID: 21142972.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    139. Flotte TR, Conlon TJ, Mueller C. Preclinical study design for rAAV. Methods Mol Biol. 2011; 807:317-37. PMID: 22034037.
      Citations: 2     Fields:    Translation:HumansCells
    140. Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, Su Q, He R, Mu X, Seher Ahmed S, Park S, Kato H, Li C, Mueller C, Mello CC, Weng Z, Flotte TR, Zamore PD, Gao G. MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther. 2011 Mar; 19(3):526-35. PMID: 21179009.
      Citations: 71     Fields:    Translation:HumansAnimalsCells
    141. Chulay JD, Ye GJ, Thomas DL, Knop DR, Benson JM, Hutt JA, Wang G, Humphries M, Flotte TR. Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther. 2011 Feb; 22(2):155-65. PMID: 20812844.
      Citations: 27     Fields:    Translation:HumansAnimalsCells
    142. Flotte TR. Sendai virus vectors. Pushing the envelope in the lung. Gene Ther. 2011 Feb; 18(2):107-8. PMID: 20927135.
      Citations:    Fields:    Translation:AnimalsCells
    143. Moore MJ, Flotte TR. Autoimmunity in a genetic disease—a cautionary tale. N Engl J Med. 2010 Oct 07; 363(15):1473-5. PMID: 20925551.
      Citations: 2     Fields:    Translation:HumansCells
    144. Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, Doria M, Douar A, Flotte TR, Francis JD, Francois A, Giacca M, Korn MT, Korytov I, Leon X, Leuchs B, Lux G, Melas C, Mizukami H, Moullier P, Müller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivière C, Roosendaal SD, Samulski RJ, Soltys SM, Surosky R, Tenenbaum L, Thomas DL, van Montfort B, Veres G, Wright JF, Xu Y, Zelenaia O, Zentilin L, Snyder RO. Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther. 2010 Oct; 21(10):1273-85. PMID: 20486768.
      Citations: 50     Fields:    Translation:Cells
    145. Mueller C, Braag SA, Keeler A, Hodges C, Drumm M, Flotte TR. Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses. Am J Respir Cell Mol Biol. 2011 Jun; 44(6):922-9. PMID: 20724552.
      Citations: 46     Fields:    Translation:AnimalsCells
    146. Martino AT, Mueller C, Braag S, Cruz PE, Campbell-Thompson M, Jin S, Flotte TR. N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance. Am J Respir Cell Mol Biol. 2011 Jun; 44(6):824-30. PMID: 20693405.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    147. Buff SM, Yu H, McCall JN, Caldwell SM, Ferkol TW, Flotte TR, Virella-Lowell IL. IL-10 delivery by AAV5 vector attenuates inflammation in mice with Pseudomonas pneumonia. Gene Ther. 2010 May; 17(5):567-76. PMID: 20357828.
      Citations: 15     Fields:    Translation:AnimalsCells
    148. Mueller C, Keeler A, Braag S, Menz T, Tang Q, Flotte TR. Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e. Mol Ther. 2010 Mar; 18(3):511-8. PMID: 19935781.
      Citations: 3     Fields:    Translation:AnimalsCells
    149. Mueller C, Strayer MS, Sirninger J, Braag S, Branco F, Louboutin JP, Flotte TR, Strayer DS. In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors. Gene Ther. 2010 Feb; 17(2):227-37. PMID: 19890354.
      Citations: 2     Fields:    Translation:AnimalsCells
    150. Flotte TR, Fischer AC, Goetzmann J, Mueller C, Cebotaru L, Yan Z, Wang L, Wilson JM, Guggino WB, Engelhardt JF. Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. Mol Ther. 2010 Mar; 18(3):594-600. PMID: 19826405.
      Citations: 32     Fields:    Translation:HumansAnimalsCells
    151. Stalvey MS, Flotte TR. Endocrine parameters of cystic fibrosis: back to basics. J Cell Biochem. 2009 Oct 01; 108(2):353-61. PMID: 19670266.
      Citations: 2     Fields:    Translation:HumansAnimals
    152. Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, Rouhani F, Conlon TJ, Calcedo R, Betts MR, Spencer C, Byrne BJ, Wilson JM, Flotte TR. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A. 2009 Sep 22; 106(38):16363-8. PMID: 19706466.
      Citations: 162     Fields:    Translation:HumansCellsCTClinical Trials
    153. Goel VK, Ibrahim N, Jiang G, Singhal M, Fee S, Flotte T, Westmoreland S, Haluska FS, Hinds PW, Haluska FG. Melanocytic nevus-like hyperplasia and melanoma in transgenic BRAFV600E mice. Oncogene. 2009 Jun 11; 28(23):2289-98. PMID: 19398955.
      Citations: 70     Fields:    Translation:HumansAnimalsCells
    154. Pashuck TD, Franz SE, Altman MK, Wasserfall CH, Atkinson MA, Wronski TJ, Flotte TR, Stalvey MS. Murine model for cystic fibrosis bone disease demonstrates osteopenia and sex-related differences in bone formation. Pediatr Res. 2009 Mar; 65(3):311-6. PMID: 19047917.
      Citations: 13     Fields:    Translation:Animals
    155. Yu H, Zeidan YH, Wu BX, Jenkins RW, Flotte TR, Hannun YA, Virella-Lowell I. Defective acid sphingomyelinase pathway with Pseudomonas aeruginosa infection in cystic fibrosis. Am J Respir Cell Mol Biol. 2009 Sep; 41(3):367-75. PMID: 19168701.
      Citations: 25     Fields:    Translation:HumansAnimalsCells
    156. Mueller C, Flotte TR. Gene therapy for cystic fibrosis. Clin Rev Allergy Immunol. 2008 Dec; 35(3):164-78. PMID: 18600482.
      Citations: 25     Fields:    Translation:HumansAnimalsCells
    157. Liqun Wang R, McLaughlin T, Cossette T, Tang Q, Foust K, Campbell-Thompson M, Martino A, Cruz P, Loiler S, Mueller C, Flotte TR. Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery. Mol Ther. 2009 Jan; 17(1):81-7. PMID: 18941444.
      Citations: 16     Fields:    Translation:AnimalsCells
    158. Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther. 2008 Oct; 19(10):979-90. PMID: 18774912.
      Citations: 485     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    159. Beattie SG, Goetzman E, Tang Q, Conlon T, Campbell-Thompson M, Matern D, Vockley J, Flotte TR. Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice. J Gene Med. 2008 Oct; 10(10):1113-23. PMID: 18720429.
      Citations: 3     Fields:    Translation:AnimalsCells
    160. Mueller C, Braag SA, Martino AT, Tang Q, Campbell-Thompson M, Flotte TR. The pros and cons of immunomodulatory IL-10 gene therapy with recombinant AAV in a Cftr-/- -dependent allergy mouse model. Gene Ther. 2009 Feb; 16(2):172-83. PMID: 18818669.
      Citations: 10     Fields:    Translation:AnimalsCells
    161. Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, Jacobson SG, Hauswirth WW. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A. 2008 Sep 30; 105(39):15112-7. PMID: 18809924.
      Citations: 359     Fields:    Translation:HumansCellsCTClinical Trials
    162. Stalvey MS, Brusko TM, Mueller C, Wasserfall CH, Schatz DA, Atkinson MA, Flotte TR. CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetes. Cytokine. 2008 Oct; 44(1):154-9. PMID: 18778952.
      Citations: 4     Fields:    Translation:AnimalsCells
    163. Silver JN, Flotte TR. Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies. Pharmacogenomics. 2008 Jul; 9(7):947-68. PMID: 18597656.
      Citations: 3     Fields:    Translation:HumansCells
    164. Flotte TR, Goetzmann J, Caridi J, Paolillo J, Conlon TJ, Potter M, Mueller C, Byrne BJ. Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees. Hum Gene Ther. 2008 Jul; 19(7):681-9. PMID: 18588426.
      Citations: 4     Fields:    Translation:AnimalsCells
    165. Beattie SG, Goetzman E, Conlon T, Germain S, Walter G, Campbell-Thompson M, Matern D, Vockley J, Flotte TR. Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD. Hum Gene Ther. 2008 Jun; 19(6):579-88. PMID: 18500942.
      Citations: 5     Fields:    Translation:AnimalsCells
    166. Cebotaru L, Vij N, Ciobanu I, Wright J, Flotte T, Guggino WB. Cystic fibrosis transmembrane regulator missing the first four transmembrane segments increases wild type and DeltaF508 processing. J Biol Chem. 2008 Aug 08; 283(32):21926-33. PMID: 18508776.
      Citations: 23     Fields:    Translation:AnimalsCells
    167. Flotte TR. In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negative. Mol Ther. 2008 May; 16(5):806-7. PMID: 18432269.
      Citations:    Fields:    Translation:HumansAnimals
    168. Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 2008 Jun; 15(11):858-63. PMID: 18418415.
      Citations: 134     Fields:    Translation:HumansAnimalsCells
    169. Foust KD, Poirier A, Pacak CA, Mandel RJ, Flotte TR. Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons. Hum Gene Ther. 2008 Jan; 19(1):61-70. PMID: 18052722.
      Citations: 36     Fields:    Translation:AnimalsCells
    170. Mueller C, Torrez D, Braag S, Martino A, Clarke T, Campbell-Thompson M, Flotte TR. Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene. J Gene Med. 2008 Jan; 10(1):51-60. PMID: 18023072.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    171. Foust KD, Flotte TR, Reier PJ, Mandel RJ. Recombinant adeno-associated virus-mediated global anterograde delivery of glial cell line-derived neurotrophic factor to the spinal cord: comparison of rubrospinal and corticospinal tracts in the rat. Hum Gene Ther. 2008 Jan; 19(1):71-82. PMID: 18072858.
      Citations: 15     Fields:    Translation:AnimalsCells
    172. Flotte TR. Gene therapy: the first two decades and the current state-of-the-art. J Cell Physiol. 2007 Nov; 213(2):301-5. PMID: 17577203.
      Citations: 28     Fields:    Translation:HumansAnimalsCells
    173. Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Ylä-Herttuala S. Progress and prospects: gene therapy clinical trials (part 1). Gene Ther. 2007 Oct; 14(20):1439-47. PMID: 17909539.
      Citations: 31     Fields:    Translation:Humans
    174. Cruz PE, Mueller C, Flotte TR. The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency. Pharmacogenomics. 2007 Sep; 8(9):1191-8. PMID: 17924834.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    175. Cruz PE, Mueller C, Cossette TL, Golant A, Tang Q, Beattie SG, Brantly M, Campbell-Thompson M, Blomenkamp KS, Teckman JH, Flotte TR. In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. Lab Invest. 2007 Sep; 87(9):893-902. PMID: 17592477.
      Citations: 24     Fields:    Translation:HumansAnimalsCells
    176. Erger KE, Conlon TJ, Leal NA, Zori R, Bobik TA, Flotte TR. In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8. J Gene Med. 2007 Jun; 9(6):462-9. PMID: 17471589.
      Citations:    Fields:    Translation:HumansAnimalsCells
    177. Chen B, Kapturczak MH, Joseph R, George JF, Campbell-Thompson M, Wasserfall CH, Atkinson MA, Tisher CC, Flotte TR, Agarwal A, Chen S. Adeno-associated viral vector-mediated interleukin-10 prolongs allograft survival in a rat kidney transplantation model. Am J Transplant. 2007 May; 7(5):1112-20. PMID: 17456199.
      Citations: 11     Fields:    Translation:AnimalsCells
    178. Flotte TR, Conlon TJ, Poirier A, Campbell-Thompson M, Byrne BJ. Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. Hum Gene Ther. 2007 Mar; 18(3):245-56. PMID: 17376008.
      Citations: 22     Fields:    Translation:HumansAnimalsCells
    179. Fischer AC, Smith CI, Cebotaru L, Zhang X, Askin FB, Wright J, Guggino SE, Adams RJ, Flotte T, Guggino WB. Expression of a truncated cystic fibrosis transmembrane conductance regulator with an AAV5-pseudotyped vector in primates. Mol Ther. 2007 Apr; 15(4):756-63. PMID: 17299412.
      Citations: 22     Fields:    Translation:AnimalsCells
    180. Flotte TR, Ng P, Dylla DE, McCray PB, Wang G, Kolls JK, Hu J. Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol Ther. 2007 Feb; 15(2):229-41. PMID: 17235299.
      Citations: 27     Fields:    Translation:HumansAnimalsCells
    181. Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther. 2006 Dec; 17(12):1177-86. PMID: 17115945.
      Citations: 73     Fields:    Translation:HumansCellsCTClinical Trials
    182. Petrache I, Fijalkowska I, Medler TR, Skirball J, Cruz P, Zhen L, Petrache HI, Flotte TR, Tuder RM. alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis. Am J Pathol. 2006 Oct; 169(4):1155-66. PMID: 17003475.
      Citations: 129     Fields:    Translation:HumansAnimalsCells
    183. Milligan ED, Sloane EM, Langer SJ, Hughes TS, Jekich BM, Frank MG, Mahoney JH, Levkoff LH, Maier SF, Cruz PE, Flotte TR, Johnson KW, Mahoney MM, Chavez RA, Leinwand LA, Watkins LR. Repeated intrathecal injections of plasmid DNA encoding interleukin-10 produce prolonged reversal of neuropathic pain. Pain. 2006 Dec 15; 126(1-3):294-308. PMID: 16949747.
      Citations: 74     Fields:    Translation:HumansAnimals
    184. Flotte TR, Frentzen B, Humphries MR, Rosenbloom AL. Recent developments in the protection of pediatric research subjects. J Pediatr. 2006 Sep; 149(3):285-286. PMID: 16939731.
      Citations: 1     Fields:    Translation:Humans
    185. Blenman KR, Duan B, Xu Z, Wan S, Atkinson MA, Flotte TR, Croker BP, Morel L. IL-10 regulation of lupus in the NZM2410 murine model. Lab Invest. 2006 Nov; 86(11):1136-48. PMID: 16924244.
      Citations: 41     Fields:    Translation:AnimalsCells
    186. Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther. 2006 Aug; 17(8):845-58. PMID: 16942444.
      Citations: 71     Fields:    Translation:HumansAnimalsCells
    187. Stalvey MS, Muller C, Schatz DA, Wasserfall CH, Campbell-Thompson ML, Theriaque DW, Flotte TR, Atkinson MA. Cystic fibrosis transmembrane conductance regulator deficiency exacerbates islet cell dysfunction after beta-cell injury. Diabetes. 2006 Jul; 55(7):1939-45. PMID: 16804061.
      Citations: 16     Fields:    Translation:Animals
    188. Lu Y, Choi YK, Campbell-Thompson M, Li C, Tang Q, Crawford JM, Flotte TR, Song S. Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector. J Gene Med. 2006 Jun; 8(6):730-5. PMID: 16518879.
      Citations: 21     Fields:    Translation:HumansAnimalsCells
    189. Jacobson SG, Acland GM, Aguirre GD, Aleman TS, Schwartz SB, Cideciyan AV, Zeiss CJ, Komaromy AM, Kaushal S, Roman AJ, Windsor EA, Sumaroka A, Pearce-Kelling SE, Conlon TJ, Chiodo VA, Boye SL, Flotte TR, Maguire AM, Bennett J, Hauswirth WW. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol Ther. 2006 Jun; 13(6):1074-84. PMID: 16644289.
      Citations: 111     Fields:    Translation:AnimalsCells
    190. Seo BB, Nakamaru-Ogiso E, Flotte TR, Matsuno-Yagi A, Yagi T. In vivo complementation of complex I by the yeast Ndi1 enzyme. Possible application for treatment of Parkinson disease. J Biol Chem. 2006 May 19; 281(20):14250-5. PMID: 16543240.
      Citations: 35     Fields:    Translation:AnimalsCells
    191. Petrache I, Fijalkowska I, Zhen L, Medler TR, Brown E, Cruz P, Choe KH, Taraseviciene-Stewart L, Scerbavicius R, Shapiro L, Zhang B, Song S, Hicklin D, Voelkel NF, Flotte T, Tuder RM. A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysema. Am J Respir Crit Care Med. 2006 Jun 01; 173(11):1222-8. PMID: 16514110.
      Citations: 88     Fields:    Translation:HumansAnimalsCells
    192. Müller C, Braag SA, Herlihy JD, Wasserfall CH, Chesrown SE, Nick HS, Atkinson MA, Flotte TR. Enhanced IgE allergic response to Aspergillus fumigatus in CFTR-/- mice. Lab Invest. 2006 Feb; 86(2):130-40. PMID: 16424881.
      Citations: 10     Fields:    Translation:Animals
    193. Conlon TJ, Walter G, Owen R, Cossette T, Erger K, Gutierrez G, Goetzman E, Matern D, Vockley J, Flotte TR. Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector. Hum Gene Ther. 2006 Jan; 17(1):71-80. PMID: 16409126.
      Citations: 8     Fields:    Translation:HumansAnimalsCells
    194. Kaplan KA, Beierle EA, Faro A, Eskin TA, Flotte TR. Recurrent pneumonia in children: a case report and approach to diagnosis. Clin Pediatr (Phila). 2006 Jan-Feb; 45(1):15-22. PMID: 16429211.
      Citations: 4     Fields:    Translation:Humans
    195. Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res. 2005 Dec; 58(6):1143-7. PMID: 16306183.
      Citations: 14     Fields:    Translation:HumansCells
    196. Loiler SA, Tang Q, Clarke T, Campbell-Thompson ML, Chiodo V, Hauswirth W, Cruz P, Perret-Gentil M, Atkinson MA, Ramiya VK, Flotte TR. Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. Mol Ther. 2005 Sep; 12(3):519-27. PMID: 15979413.
      Citations: 14     Fields:    Translation:AnimalsCells
    197. Swiatecka-Urban A, Brown A, Moreau-Marquis S, Renuka J, Coutermarsh B, Barnaby R, Karlson KH, Flotte TR, Fukuda M, Langford GM, Stanton BA. The short apical membrane half-life of rescued {Delta}F508-cystic fibrosis transmembrane conductance regulator (CFTR) results from accelerated endocytosis of {Delta}F508-CFTR in polarized human airway epithelial cells. J Biol Chem. 2005 Nov 04; 280(44):36762-72. PMID: 16131493.
      Citations: 111     Fields:    Translation:HumansCells
    198. Conlon TJ, Cossette T, Erger K, Choi YK, Clarke T, Scott-Jorgensen M, Song S, Campbell-Thompson M, Crawford J, Flotte TR. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther. 2005 Nov; 12(5):867-75. PMID: 16085464.
      Citations: 24     Fields:    Translation:HumansAnimalsCells
    199. Flotte TR, Schwiebert EM, Zeitlin PL, Carter BJ, Guggino WB. Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy. Hum Gene Ther. 2005 Aug; 16(8):921-8. PMID: 16076250.
      Citations: 12     Fields:    Translation:HumansCellsCTClinical Trials
    200. Virella-Lowell I, Zusman B, Foust K, Loiler S, Conlon T, Song S, Chesnut KA, Ferkol T, Flotte TR. Enhancing rAAV vector expression in the lung. J Gene Med. 2005 Jul; 7(7):842-50. PMID: 15838934.
      Citations: 27     Fields:    Translation:HumansAnimalsCells
    201. Flotte TR. Recent developments in recombinant AAV-mediated gene therapy for lung diseases. Curr Gene Ther. 2005 Jun; 5(3):361-6. PMID: 15975013.
      Citations: 19     Fields:    Translation:HumansAnimalsCells
    202. Flotte TR. Adeno-associated virus-mediated gene transfer for lung diseases. Hum Gene Ther. 2005 Jun; 16(6):643-8. PMID: 15960596.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    203. Chen S, Kapturczak MH, Wasserfall C, Glushakova OY, Campbell-Thompson M, Deshane JS, Joseph R, Cruz PE, Hauswirth WW, Madsen KM, Croker BP, Berns KI, Atkinson MA, Flotte TR, Tisher CC, Agarwal A. Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway. Proc Natl Acad Sci U S A. 2005 May 17; 102(20):7251-6. PMID: 15878989.
      Citations: 46     Fields:    Translation:AnimalsCells
    204. Flotte TR, Berns KI. Adeno-associated virus: a ubiquitous commensal of mammals. Hum Gene Ther. 2005 Apr; 16(4):401-7. PMID: 15871671.
      Citations: 21     Fields:    Translation:HumansAnimalsCells
    205. Milligan ED, Sloane EM, Langer SJ, Cruz PE, Chacur M, Spataro L, Wieseler-Frank J, Hammack SE, Maier SF, Flotte TR, Forsayeth JR, Leinwand LA, Chavez R, Watkins LR. Controlling neuropathic pain by adeno-associated virus driven production of the anti-inflammatory cytokine, interleukin-10. Mol Pain. 2005 Feb 25; 1:9. PMID: 15813997.
      Citations: 85     Fields:    Translation:HumansAnimalsCells
    206. Mohiuddin I, Loiler S, Zolotukhin I, Byrne BJ, Flotte TR, Snyder RO. Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Mol Ther. 2005 Feb; 11(2):320-6. PMID: 15668144.
      Citations: 6     Fields:    Translation:HumansAnimalsCells
    207. Chen S, Kapturczak M, Loiler SA, Zolotukhin S, Glushakova OY, Madsen KM, Samulski RJ, Hauswirth WW, Campbell-Thompson M, Berns KI, Flotte TR, Atkinson MA, Tisher CC, Agarwal A. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum Gene Ther. 2005 Feb; 16(2):235-47. PMID: 15761263.
      Citations: 33     Fields:    Translation:HumansAnimalsCells
    208. Song S, Witek RP, Lu Y, Choi YK, Zheng D, Jorgensen M, Li C, Flotte TR, Petersen BE. Ex vivo transduced liver progenitor cells as a platform for gene therapy in mice. Hepatology. 2004 Oct; 40(4):918-24. PMID: 15382177.
      Citations: 16     Fields:    Translation:AnimalsCells
    209. Sirninger J, Muller C, Braag S, Tang Q, Yue H, Detrisac C, Ferkol T, Guggino WB, Flotte TR. Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector. Hum Gene Ther. 2004 Sep; 15(9):832-41. PMID: 15353038.
      Citations: 34     Fields:    Translation:AnimalsCells
    210. Seo BB, Nakamaru-Ogiso E, Cruz P, Flotte TR, Yagi T, Matsuno-Yagi A. Functional expression of the single subunit NADH dehydrogenase in mitochondria in vivo: a potential therapy for complex I deficiencies. Hum Gene Ther. 2004 Sep; 15(9):887-95. PMID: 15353043.
      Citations: 14     Fields:    Translation:AnimalsCells
    211. Virella-Lowell I, Herlihy JD, Liu B, Lopez C, Cruz P, Muller C, Baker HV, Flotte TR. Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line. Mol Ther. 2004 Sep; 10(3):562-73. PMID: 15336656.
      Citations: 21     Fields:    Translation:HumansAnimalsCells
    212. Flotte TR. Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective. Hum Gene Ther. 2004 Jul; 15(7):716-7. PMID: 15242531.
      Citations: 5     Fields:    Translation:HumansAnimalsCells
    213. Conlon TJ, Flotte TR. Recombinant adeno-associated virus vectors for gene therapy. Expert Opin Biol Ther. 2004 Jul; 4(7):1093-101. PMID: 15268676.
      Citations: 15     Fields:    Translation:HumansAnimalsCells
    214. Flotte TR. Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2004 May; 11(10):805-10. PMID: 15042119.
      Citations: 39     Fields:    Translation:HumansCells
    215. Bray CL, Cahill KS, Oshier JT, Peden CS, Theriaque DW, Flotte TR, Stacpoole PW. Methylphenidate does not improve cognitive function in healthy sleep-deprived young adults. J Investig Med. 2004 Apr; 52(3):192-201. PMID: 15222409.
      Citations: 5     Fields:    Translation:HumansCTClinical Trials
    216. Song S, Lu Y, Choi YK, Han Y, Tang Q, Zhao G, Berns KI, Flotte TR. DNA-dependent PK inhibits adeno-associated virus DNA integration. Proc Natl Acad Sci U S A. 2004 Feb 17; 101(7):2112-6. PMID: 14766968.
      Citations: 47     Fields:    Translation:HumansAnimalsCells
    217. Zhang YC, Powers M, Wasserfall C, Brusko T, Song S, Flotte T, Snyder RO, Potter M, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Nick HS, Agarwal A, Ellis TM, Atkinson MA. Immunity to adeno-associated virus serotype 2 delivered transgenes imparted by genetic predisposition to autoimmunity. Gene Ther. 2004 Feb; 11(3):233-40. PMID: 14737082.
      Citations: 5     Fields:    Translation:AnimalsCells
    218. Song S, Goudy K, Campbell-Thompson M, Wasserfall C, Scott-Jorgensen M, Wang J, Tang Q, Crawford JM, Ellis TM, Atkinson MA, Flotte TR. Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice. Gene Ther. 2004 Jan; 11(2):181-6. PMID: 14712302.
      Citations: 39     Fields:    Translation:HumansAnimalsCells
    219. Flotte TR, Brantly ML, Spencer LT, Byrne BJ, Spencer CT, Baker DJ, Humphries M. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther. 2004 Jan; 15(1):93-128. PMID: 14965381.
      Citations: 50     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    220. Fischer AC, Beck SE, Smith CI, Laube BL, Askin FB, Guggino SE, Adams RJ, Flotte TR, Guggino WB. Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques. Mol Ther. 2003 Dec; 8(6):918-26. PMID: 14664794.
      Citations: 12     Fields:    Translation:AnimalsCells
    221. Burkhardt BR, Loiler SA, Anderson JA, Kilberg MS, Crawford JM, Flotte TR, Goudy KS, Ellis TM, Atkinson M. Glucose-responsive expression of the human insulin promoter in HepG2 human hepatoma cells. Ann N Y Acad Sci. 2003 Nov; 1005:237-41. PMID: 14679068.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    222. Loiler SA, Conlon TJ, Song S, Tang Q, Warrington KH, Agarwal A, Kapturczak M, Li C, Ricordi C, Atkinson MA, Muzyczka N, Flotte TR. Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver. Gene Ther. 2003 Sep; 10(18):1551-8. PMID: 12907946.
      Citations: 32     Fields:    Translation:HumansAnimalsCells
    223. Goudy KS, Burkhardt BR, Wasserfall C, Song S, Campbell-Thompson ML, Brusko T, Powers MA, Clare-Salzler MJ, Sobel ES, Ellis TM, Flotte TR, Atkinson MA. Systemic overexpression of IL-10 induces CD4+CD25+ cell populations in vivo and ameliorates type 1 diabetes in nonobese diabetic mice in a dose-dependent fashion. J Immunol. 2003 Sep 01; 171(5):2270-8. PMID: 12928371.
      Citations: 36     Fields:    Translation:AnimalsCells
    224. Flotte TR, Zeitlin PL, Reynolds TC, Heald AE, Pedersen P, Beck S, Conrad CK, Brass-Ernst L, Humphries M, Sullivan K, Wetzel R, Taylor G, Carter BJ, Guggino WB. Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther. 2003 Jul 20; 14(11):1079-88. PMID: 12885347.
      Citations: 62     Fields:    Translation:HumansCellsCTClinical Trials
    225. Stacpoole PW, Owen R, Flotte TR. The pyruvate dehydrogenase complex as a target for gene therapy. Curr Gene Ther. 2003 Jun; 3(3):239-45. PMID: 12762482.
      Citations: 6     Fields:    Translation:HumansAnimalsCells
    226. Flotte TR. The signal and the trap: targeted delivery and retention of proteins in the mitochondrion. Mol Ther. 2003 Jun; 7(6):715-6. PMID: 12803173.
      Citations:    Fields:    Translation:HumansAnimalsCells
    227. Chen S, Agarwal A, Glushakova OY, Jorgensen MS, Salgar SK, Poirier A, Flotte TR, Croker BP, Madsen KM, Atkinson MA, Hauswirth WW, Berns KI, Tisher CC. Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors. J Am Soc Nephrol. 2003 Apr; 14(4):947-58. PMID: 12660329.
      Citations: 17     Fields:    Translation:AnimalsCells
    228. Zhang YC, Pileggi A, Agarwal A, Molano RD, Powers M, Brusko T, Wasserfall C, Goudy K, Zahr E, Poggioli R, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Nick H, Flotte T, Ellis TM, Ricordi C, Inverardi L, Atkinson MA. Adeno-associated virus-mediated IL-10 gene therapy inhibits diabetes recurrence in syngeneic islet cell transplantation of NOD mice. Diabetes. 2003 Mar; 52(3):708-16. PMID: 12606512.
      Citations: 27     Fields:    Translation:AnimalsCellsPHPublic Health
    229. Barbour KW, Wei F, Brannan C, Flotte TR, Baumann H, Berger FG. The murine alpha(1)-proteinase inhibitor gene family: polymorphism, chromosomal location, and structure. Genomics. 2002 Nov; 80(5):515-22. PMID: 12408969.
      Citations: 5     Fields:    Translation:AnimalsCells
    230. Zhang YC, Molano RD, Pileggi A, Powers M, Cross J, Wasserfall C, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Flotte T, Ellis TM, Ricordi C, Atkinson MA, Inverardi L. Adeno-associated virus transduction of islets with interleukin-4 results in impaired metabolic function in syngeneic marginal islet mass transplantation. Transplantation. 2002 Oct 27; 74(8):1184-6. PMID: 12438968.
      Citations: 1     Fields:    Translation:AnimalsCells
    231. Beck SE, Laube BL, Barberena CI, Fischer AC, Adams RJ, Chesnut K, Flotte TR, Guggino WB. Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques. Mol Ther. 2002 Oct; 6(4):546-54. PMID: 12387250.
      Citations: 28     Fields:    Translation:AnimalsCells
    232. Snyder RO, Flotte TR. Production of clinical-grade recombinant adeno-associated virus vectors. Curr Opin Biotechnol. 2002 Oct; 13(5):418-23. PMID: 12459331.
      Citations: 18     Fields:    Translation:HumansAnimalsCells
    233. Song S, Scott-Jorgensen M, Wang J, Poirier A, Crawford J, Campbell-Thompson M, Flotte TR. Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects. Mol Ther. 2002 Sep; 6(3):329-35. PMID: 12231168.
      Citations: 24     Fields:    Translation:AnimalsCells
    234. Seo BB, Nakamaru-Ogiso E, Flotte TR, Yagi T, Matsuno-Yagi A. A single-subunit NADH-quinone oxidoreductase renders resistance to mammalian nerve cells against complex I inhibition. Mol Ther. 2002 Sep; 6(3):336-41. PMID: 12231169.
      Citations: 18     Fields:    Translation:HumansAnimalsCells
    235. Owen R, Mandel RJ, Ammini CV, Conlon TJ, Kerr DS, Stacpoole PW, Flotte TR. Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectors. Mol Ther. 2002 Sep; 6(3):394-9. PMID: 12231176.
      Citations: 2     Fields:    Translation:HumansCells
    236. Wagner JA, Nepomuceno IB, Messner AH, Moran ML, Batson EP, Dimiceli S, Brown BW, Desch JK, Norbash AM, Conrad CK, Guggino WB, Flotte TR, Wine JJ, Carter BJ, Reynolds TC, Moss RB, Gardner P. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther. 2002 Jul 20; 13(11):1349-59. PMID: 12162817.
      Citations: 75     Fields:    Translation:HumansCellsPHPublic HealthCTClinical Trials
    237. Mah C, Fraites TJ, Zolotukhin I, Song S, Flotte TR, Dobson J, Batich C, Byrne BJ. Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther. 2002 Jul; 6(1):106-12. PMID: 12095310.
      Citations: 45     Fields:    Translation:HumansAnimalsCells
    238. Flotte TR. Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest. 2002 Mar; 121(3 Suppl):98S-102S. PMID: 11893723.
      Citations: 7     Fields:    Translation:HumansAnimalsCells
    239. Kapturczak M, Zolotukhin S, Cross J, Pileggi A, Molano RD, Jorgensen M, Byrne B, Flotte TR, Ellis T, Inverardi L, Ricordi C, Nick H, Atkinson M, Agarwal A. Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. Mol Ther. 2002 Feb; 5(2):154-60. PMID: 11829522.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    240. Mah C, Byrne BJ, Flotte TR. Virus-based gene delivery systems. Clin Pharmacokinet. 2002; 41(12):901-11. PMID: 12222993.
      Citations: 14     Fields:    Translation:HumansAnimalsCells
    241. Flotte TR, Virella-Lowell I, Chesnut KA. Adeno-associated viral vectors for CF gene therapy. Methods Mol Med. 2002; 70:599-608. PMID: 11917554.
      Citations:    Fields:    Translation:HumansCells
    242. Goudy K, Song S, Wasserfall C, Zhang YC, Kapturczak M, Muir A, Powers M, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Ellis TM, Flotte TR, Atkinson MA. Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD mice. Proc Natl Acad Sci U S A. 2001 Nov 20; 98(24):13913-8. PMID: 11717448.
      Citations: 36     Fields:    Translation:HumansAnimalsCells
    243. Flotte TR. Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Curr Opin Mol Ther. 2001 Oct; 3(5):497-502. PMID: 11699895.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    244. Donsante A, Vogler C, Muzyczka N, Crawford JM, Barker J, Flotte T, Campbell-Thompson M, Daly T, Sands MS. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther. 2001 Sep; 8(17):1343-6. PMID: 11571571.
      Citations: 86     Fields:    Translation:AnimalsCells
    245. Flotte TR, Laube BL. Gene therapy in cystic fibrosis. Chest. 2001 Sep; 120(3 Suppl):124S-131S. PMID: 11555567.
      Citations: 24     Fields:    Translation:HumansAnimalsCells
    246. Song S, Embury J, Laipis PJ, Berns KI, Crawford JM, Flotte TR. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2001 Sep; 8(17):1299-306. PMID: 11571566.
      Citations: 49     Fields:    Translation:HumansAnimalsCells
    247. Owen R, Flotte TR. Approaches and limitations to gene therapy for mitochondrial diseases. Antioxid Redox Signal. 2001 Jun; 3(3):451-60. PMID: 11491656.
      Citations:    Fields:    Translation:HumansAnimalsCells
    248. Kapturczak MH, Flotte T, Atkinson MA. Adeno-associated virus (AAV) as a vehicle for therapeutic gene delivery: improvements in vector design and viral production enhance potential to prolong graft survival in pancreatic islet cell transplantation for the reversal of type 1 diabetes. Curr Mol Med. 2001 May; 1(2):245-58. PMID: 11899074.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    249. Xu L, Daly T, Gao C, Flotte TR, Song S, Byrne BJ, Sands MS, Parker Ponder K. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Hum Gene Ther. 2001 Mar 20; 12(5):563-73. PMID: 11268288.
      Citations: 53     Fields:    Translation:HumansAnimalsCells
    250. Song S, Laipis PJ, Berns KI, Flotte TR. Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle. Proc Natl Acad Sci U S A. 2001 Mar 27; 98(7):4084-8. PMID: 11274433.
      Citations: 42     Fields:    Translation:AnimalsCells
    251. Flotte T, Agarwal A, Wang J, Song S, Fenjves ES, Inverardi L, Chesnut K, Afione S, Loiler S, Wasserfall C, Kapturczak M, Ellis T, Nick H, Atkinson M. Efficient ex vivo transduction of pancreatic islet cells with recombinant adeno-associated virus vectors. Diabetes. 2001 Mar; 50(3):515-20. PMID: 11246870.
      Citations: 18     Fields:    Translation:HumansAnimalsCells
    252. Stacpoole PW, Fisher WR, Flotte TR, Geiser EA, Theriaque DW, Hutson AD. Teaching hypothesis-oriented thinking to medical students: the University of Florida's clinical investigation program. Acad Med. 2001 Mar; 76(3):287-92. PMID: 11242585.
      Citations: 2     Fields:    Translation:Humans
    253. Seo BB, Wang J, Flotte TR, Yagi T, Matsuno-Yagi A. Use of the NADH-quinone oxidoreductase (NDI1) gene of Saccharomyces cerevisiae as a possible cure for complex I defects in human cells. J Biol Chem. 2000 Dec 01; 275(48):37774-8. PMID: 10982813.
      Citations: 31     Fields:    Translation:HumansCells
    254. Fishbein L, O'Brien P, Hutson A, Theriaque D, Stacpoole PW, Flotte T. Pharmacokinetics and pharmacodynamic effects of nicotine nasal spray devices on cardiovascular and pulmonary function. J Investig Med. 2000 Nov; 48(6):435-40. PMID: 11094866.
      Citations: 4     Fields:    Translation:HumansCTClinical Trials
    255. Owen R IV, Lewin AP, Peel A, Wang J, Guy J, Hauswirth WW, Stacpoole PW, Flotte TR. Recombinant adeno-associated virus vector-based gene transfer for defects in oxidative metabolism. Hum Gene Ther. 2000 Oct 10; 11(15):2067-78. PMID: 11044909.
      Citations: 8     Fields:    Translation:HumansAnimalsCells
    256. Virella-Lowell I, Poirier A, Chesnut KA, Brantly M, Flotte TR. Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther. 2000 Oct; 7(20):1783-9. PMID: 11083501.
      Citations: 28     Fields:    Translation:HumansCells
    257. Wu P, Xiao W, Conlon T, Hughes J, Agbandje-McKenna M, Ferkol T, Flotte T, Muzyczka N. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol. 2000 Sep; 74(18):8635-47. PMID: 10954565.
      Citations: 145     Fields:    Translation:HumansCells
    258. Flotte TR. Size does matter: overcoming the adeno-associated virus packaging limit. Respir Res. 2000; 1(1):16-8. PMID: 11667959.
      Citations: 6     Fields:    Translation:AnimalsCells
    259. Beck SE, Jones LA, Chesnut K, Walsh SM, Reynolds TC, Carter BJ, Askin FB, Flotte TR, Guggino WB. Repeated delivery of adeno-associated virus vectors to the rabbit airway. J Virol. 1999 Nov; 73(11):9446-55. PMID: 10516053.
      Citations: 31     Fields:    Translation:AnimalsCells
    260. Hernandez YJ, Wang J, Kearns WG, Loiler S, Poirier A, Flotte TR. Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model. J Virol. 1999 Oct; 73(10):8549-58. PMID: 10482608.
      Citations: 51     Fields:    Translation:AnimalsCells
    261. Flotte TR. Gene therapy for cystic fibrosis. Curr Opin Mol Ther. 1999 Aug; 1(4):510-6. PMID: 11713767.
      Citations: 7     Fields:    Translation:HumansAnimalsCells
    262. Wagner JA, Messner AH, Moran ML, Daifuku R, Kouyama K, Desch JK, Manley S, Norbash AM, Conrad CK, Friborg S, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope. 1999 Feb; 109(2 Pt 1):266-74. PMID: 10890777.
      Citations: 57     Fields:    Translation:HumansCellsCTClinical Trials
    263. Afione SA, Wang J, Walsh S, Guggino WB, Flotte TR. Delayed expression of adeno-associated virus vector DNA. Intervirology. 1999; 42(4):213-20. PMID: 10567839.
      Citations: 8     Fields:    Translation:HumansCells
    264. Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A. 1998 Nov 24; 95(24):14384-8. PMID: 9826709.
      Citations: 92     Fields:    Translation:HumansAnimalsCells
    265. Wagner JA, Reynolds T, Moran ML, Moss RB, Wine JJ, Flotte TR, Gardner P. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet. 1998 Jun 06; 351(9117):1702-3. PMID: 9734891.
      Citations: 62     Fields:    Translation:HumansCells
    266. Wagner JA, Moran ML, Messner AH, Daifuku R, Conrad CK, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum Gene Ther. 1998 Apr 10; 9(6):889-909. PMID: 9581911.
      Citations: 28     Fields:    Translation:HumansCellsCTClinical Trials
    267. Flotte TR, Beck SE, Chesnut K, Potter M, Poirier A, Zolotukhin S. A fluorescence video-endoscopy technique for detection of gene transfer and expression. Gene Ther. 1998 Feb; 5(2):166-73. PMID: 9578835.
      Citations: 8     Fields:    Translation:AnimalsCells
    268. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol. 1998; 292:717-32. PMID: 9711594.
      Citations: 11     Fields:    Translation:HumansAnimalsCells
    269. Rubenstein RC, McVeigh U, Flotte TR, Guggino WB, Zeitlin PL. CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector. Gene Ther. 1997 May; 4(5):384-92. PMID: 9274714.
      Citations: 17     Fields:    Translation:AnimalsCells
    270. Flotte TR, Ferkol TW. Genetic therapy. Past, present, and future. Pediatr Clin North Am. 1997 Feb; 44(1):153-78. PMID: 9057789.
      Citations: 3     Fields:    Translation:Humans
    271. Flotte TR, Carter BJ. In vivo gene therapy with adeno-associated virus vectors for cystic fibrosis. Adv Pharmacol. 1997; 40:85-101. PMID: 9217924.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    272. Kearns WG, Afione SA, Fulmer SB, Pang MC, Erikson D, Egan M, Landrum MJ, Flotte TR, Cutting GR. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther. 1996 Sep; 3(9):748-55. PMID: 8875221.
      Citations: 51     Fields:    Translation:HumansCells
    273. Conrad CK, Allen SS, Afione SA, Reynolds TC, Beck SE, Fee-Maki M, Barrazza-Ortiz X, Adams R, Askin FB, Carter BJ, Guggino WB, Flotte TR. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther. 1996 Aug; 3(8):658-68. PMID: 8854091.
      Citations: 48     Fields:    Translation:AnimalsCells
    274. Baudard M, Flotte TR, Aran JM, Thierry AR, Pastan I, Pang MG, Kearns WG, Gottesman MM. Expression of the human multidrug resistance and glucocerebrosidase cDNAs from adeno-associated vectors: efficient promoter activity of AAV sequences and in vivo delivery via liposomes. Hum Gene Ther. 1996 Jul 10; 7(11):1309-22. PMID: 8818718.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    275. Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, Taylor G, Walden S, Wetzel R. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther. 1996 Jun 10; 7(9):1145-59. PMID: 8773517.
      Citations: 77     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    276. Afione SA, Conrad CK, Kearns WG, Chunduru S, Adams R, Reynolds TC, Guggino WB, Cutting GR, Carter BJ, Flotte TR. In vivo model of adeno-associated virus vector persistence and rescue. J Virol. 1996 May; 70(5):3235-41. PMID: 8627804.
      Citations: 63     Fields:    Translation:HumansAnimalsCells
    277. Carter BJ, Flotte TR. Development of adeno-associated virus vectors for gene therapy of cystic fibrosis. Curr Top Microbiol Immunol. 1996; 218:119-44. PMID: 8794249.
      Citations: 9     Fields:    Translation:HumansAnimalsCells
    278. Zeitlin PL, Chu S, Conrad C, McVeigh U, Ferguson K, Flotte TR, Guggino WB. Alveolar stem cell transduction by an adeno-associated viral vector. Gene Ther. 1995 Nov; 2(9):623-31. PMID: 8548551.
      Citations: 9     Fields:    Translation:AnimalsCells
    279. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy. Gene Ther. 1995 Aug; 2(6):357-62. PMID: 7584109.
      Citations: 75     Fields:    Translation:HumansAnimalsCells
    280. Murray CB, Morales MM, Flotte TR, McGrath-Morrow SA, Guggino WB, Zeitlin PL. CIC-2: a developmentally dependent chloride channel expressed in the fetal lung and downregulated after birth. Am J Respir Cell Mol Biol. 1995 Jun; 12(6):597-604. PMID: 7766424.
      Citations: 34     Fields:    Translation:AnimalsCells
    281. Carroll TP, Morales MM, Fulmer SB, Allen SS, Flotte TR, Cutting GR, Guggino WB. Alternate translation initiation codons can create functional forms of cystic fibrosis transmembrane conductance regulator. J Biol Chem. 1995 May 19; 270(20):11941-6. PMID: 7538127.
      Citations: 24     Fields:    Translation:HumansAnimalsCells
    282. Afione SA, Conrad CK, Flotte TR. Gene therapy vectors as drug delivery systems. Clin Pharmacokinet. 1995 Mar; 28(3):181-9. PMID: 7758249.
      Citations: 4     Fields:    Translation:Humans
    283. Flotte TR, Barraza-Ortiz X, Solow R, Afione SA, Carter BJ, Guggino WB. An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther. 1995 Jan; 2(1):29-37. PMID: 7712331.
      Citations: 24     Fields:    Translation:AnimalsCells
    284. Flotte TR, Afione SA, Zeitlin PL. Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol. 1994 Nov; 11(5):517-21. PMID: 7946381.
      Citations: 50     Fields:    Translation:HumansCells
    285. Schwiebert EM, Flotte T, Cutting GR, Guggino WB. Both CFTR and outwardly rectifying chloride channels contribute to cAMP-stimulated whole cell chloride currents. Am J Physiol. 1994 May; 266(5 Pt 1):C1464-77. PMID: 7515570.
      Citations: 33     Fields:    Translation:HumansCells
    286. Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci U S A. 1993 Nov 15; 90(22):10613-7. PMID: 7504271.
      Citations: 132     Fields:    Translation:HumansAnimalsCells
    287. Tomares SM, Flotte TR, Tunkel DE, Pao M, Loughlin GM. Real time laryngoscopy with olfactory challenge for diagnosis of psychogenic stridor. Pediatr Pulmonol. 1993 Oct; 16(4):259-62. PMID: 8265275.
      Citations: 2     Fields:    Translation:Humans
    288. Flotte TR, Afione SA, Solow R, Drumm ML, Markakis D, Guggino WB, Zeitlin PL, Carter BJ. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem. 1993 Feb 15; 268(5):3781-90. PMID: 7679117.
      Citations: 126     Fields:    Translation:HumansCells
    289. Flotte TR. Prospects for virus-based gene therapy for cystic fibrosis. J Bioenerg Biomembr. 1993 Feb; 25(1):37-42. PMID: 8382676.
      Citations: 3     Fields:    Translation:HumansCells
    290. Bhatta N, Isaacson K, Flotte T, Schiff I, Anderson RR. Injury and adhesion formation following ovarian wedge resection with different thermal surgical modalities. Lasers Surg Med. 1993; 13(3):344-52. PMID: 8515673.
      Citations: 2     Fields:    Translation:Animals
    291. Flotte TR, Solow R, Owens RA, Afione S, Zeitlin PL, Carter BJ. Gene expression from adeno-associated virus vectors in airway epithelial cells. Am J Respir Cell Mol Biol. 1992 Sep; 7(3):349-56. PMID: 1325813.
      Citations: 40     Fields:    Translation:HumansCells
    292. Egan M, Flotte T, Afione S, Solow R, Zeitlin PL, Carter BJ, Guggino WB. Defective regulation of outwardly rectifying Cl- channels by protein kinase A corrected by insertion of CFTR. Nature. 1992 Aug 13; 358(6387):581-4. PMID: 1380129.
      Citations: 91     Fields:    Translation:HumansCells
    293. Husain Z, Pathak MA, Flotte T, Wick MM. Role of ultraviolet radiation in the induction of melanocytic tumors in hairless mice following 7,12-dimethylbenz(a)anthracene application and ultraviolet irradiation. Cancer Res. 1991 Sep 15; 51(18):4964-70. PMID: 1909931.
      Citations: 12     Fields:    Translation:AnimalsCells
    294. Flotte TR, Loughlin GM. Benefits and complications of troleandomycin (TAO) in young children with steroid-dependent asthma. Pediatr Pulmonol. 1991; 10(3):178-82. PMID: 1852515.
      Citations: 7     Fields:    Translation:Humans
    295. Flotte TR. Dietl syndrome: intermittent ureteropelvic junction obstruction as a cause of episodic abdominal pain. Pediatrics. 1988 Nov; 82(5):792-4. PMID: 3186362.
      Citations: 1     Fields:    Translation:Humans
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