Terence Flotte to Genetic Therapy
This is a "connection" page, showing publications Terence Flotte has written about Genetic Therapy.
Connection Strength
31.274
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Wang D, Stevens G, Flotte TR. Gene therapy then and now: A look back at changes in the field over the past 25 years. Mol Ther. 2025 May 07; 33(5):1889-1902.
Score: 0.591
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Daci R, Gray-Edwards H, Shazeeb MS, Vardar Z, Vachha B, Cataltepe OI, Flotte TR. Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases. Hum Gene Ther. 2024 Nov; 35(21-22):886-895.
Score: 0.578
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Flotte TR. Intrathecal gene therapy for neurologic disease in humans. Mol Ther. 2024 May 01; 32(5):1185-1186.
Score: 0.557
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Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista AR, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon CK, Lidov H, Woodman K, Pajusalu S, Spinazzola JM, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. N Engl J Med. 2023 09 28; 389(13):1203-1210.
Score: 0.535
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Chan YK, Flotte TR. Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies. Mol Ther. 2023 04 05; 31(4):913-914.
Score: 0.516
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Flotte TR. ESGCT 2022: The New Normal for the Gene Therapy Community. Hum Gene Ther. 2022 09; 33(17-18):841.
Score: 0.497
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Darukhanavala A, Flotte TR. Gene Therapy for Endocrine Disorders: A Promising Intervention. Hum Gene Ther. 2022 08; 33(15-16):780-781.
Score: 0.494
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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.
Score: 0.486
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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.
Score: 0.440
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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.
Score: 0.428
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Flotte TR, Adair JE. Black Lives Matter to Gene Therapy. Hum Gene Ther. 2020 07; 31(13-14):700.
Score: 0.428
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Flotte TR. Barrie J. Carter: The Science and the Scientist. Hum Gene Ther. 2020 05; 31(9-10):489-490.
Score: 0.423
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Flotte TR. Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors. Hum Gene Ther. 2020 04; 31(7-8):398-399.
Score: 0.420
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Flotte TR. Impact on Women's Health: Gene Therapy in Gynecologic Oncology. Hum Gene Ther. 2020 03; 31(5-6):271-272.
Score: 0.418
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Flotte TR. Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy. Hum Gene Ther. 2020 01; 31(1-2):12-13.
Score: 0.413
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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.
Score: 0.411
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Flotte TR. Epigenome Editing Strategies for Low Back Pain. Hum Gene Ther. 2019 09; 30(9):1037-1038.
Score: 0.404
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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.
Score: 0.399
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Flotte TR. Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing. Hum Gene Ther. 2019 07; 30(7):793.
Score: 0.399
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Barzel A, Flotte TR. New Horizons for Immune Gene Therapy. Hum Gene Ther. 2019 04; 30(4):379-380.
Score: 0.392
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Flotte TR. Top Five Gene Therapy Stories of 2019. Hum Gene Ther. 2019 01; 30(1):1-2.
Score: 0.386
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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.
Score: 0.366
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Gruntman AM, Flotte TR. The rapidly evolving state of gene therapy. FASEB J. 2018 04; 32(4):1733-1740.
Score: 0.366
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Flotte TR, B?ning 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.
Score: 0.362
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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.
Score: 0.362
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Flotte TR. Therapeutic Advances in Germany and Beyond. Hum Gene Ther. 2017 12; 28(12):1117.
Score: 0.358
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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.
Score: 0.357
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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.
Score: 0.356
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Keeler AM, ElMallah MK, Flotte TR. Gene Therapy 2017: Progress and Future Directions. Clin Transl Sci. 2017 07; 10(4):242-248.
Score: 0.345
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Flotte TR, Gao G. AAV Is Now a Medicine: We Had Better Get This Right. Hum Gene Ther. 2017 04; 28(4):307.
Score: 0.341
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Flotte TR. The Negative Effects of Immigration Restrictions on the Gene Therapy Community. Hum Gene Ther. 2017 03; 28(3):227.
Score: 0.339
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Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275.
Score: 0.336
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Naldini L, Parker GC, Flotte TR. The Renaissance of Gene and Cell Therapy: Florence 2016. Hum Gene Ther. 2016 10; 27(10):727-728.
Score: 0.330
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Flotte TR. Gene Drives: Biological Shield or Ecological Menace? Hum Gene Ther. 2016 08; 27(8):561-2.
Score: 0.326
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Flotte TR. Why Human Gene Therapy Scientists Should Care About Model Organisms. Hum Gene Ther. 2016 May; 27(5):339-40.
Score: 0.320
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Flotte TR. A Gene Therapy Scientist's Life Well-Lived. Hum Gene Ther. 2016 Apr; 27(4):279.
Score: 0.319
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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.
Score: 0.316
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Flotte TR. Special Note from the Editor-in-Chief. Hum Gene Ther. 2016 Feb; 27(2):95.
Score: 0.315
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Flotte TR. The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy. Hum Gene Ther. 2015 Dec; 26(12):782.
Score: 0.311
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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.
Score: 0.306
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Flotte TR. Ethical Implications of the Cost of Molecularly Targeted Therapies. Hum Gene Ther. 2015 Sep; 26(9):573-4.
Score: 0.306
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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.
Score: 0.301
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Flotte TR. Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question? Hum Gene Ther. 2015 May; 26(5):245-6.
Score: 0.299
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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.
Score: 0.297
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Loring HS, Flotte TR. Current status of gene therapy for a-1 antitrypsin deficiency. Expert Opin Biol Ther. 2015 Mar; 15(3):329-36.
Score: 0.289
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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.
Score: 0.282
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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.
Score: 0.276
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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.
Score: 0.270
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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.
Score: 0.269
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Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9.
Score: 0.257
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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.
Score: 0.244
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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.
Score: 0.240
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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.
Score: 0.234
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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 a1-antitrypsin: interim results. Hum Gene Ther. 2011 Oct; 22(10):1239-47.
Score: 0.232
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Flotte TR, Mueller C. Gene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011 Apr 15; 20(R1):R87-92.
Score: 0.226
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Flotte TR, Conlon TJ, Mueller C. Preclinical study design for rAAV. Methods Mol Biol. 2011; 807:317-37.
Score: 0.221
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Moore MJ, Flotte TR. Autoimmunity in a genetic disease?a cautionary tale. N Engl J Med. 2010 Oct 07; 363(15):1473-5.
Score: 0.218
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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.
Score: 0.205
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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.
Score: 0.201
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Mueller C, Flotte TR. Gene therapy for cystic fibrosis. Clin Rev Allergy Immunol. 2008 Dec; 35(3):164-78.
Score: 0.192
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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.
Score: 0.189
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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.
Score: 0.186
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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.
Score: 0.185
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Flotte TR. In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negative. Mol Ther. 2008 May; 16(5):806-7.
Score: 0.184
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Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 2008 Jun; 15(11):858-63.
Score: 0.184
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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.
Score: 0.180
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Flotte TR. Gene therapy: the first two decades and the current state-of-the-art. J Cell Physiol. 2007 Nov; 213(2):301-5.
Score: 0.178
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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.
Score: 0.176
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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.
Score: 0.173
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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.
Score: 0.169
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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.
Score: 0.167
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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.
Score: 0.157
-
Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res. 2005 Dec; 58(6):1143-7.
Score: 0.156
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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.
Score: 0.152
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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.
Score: 0.152
-
Flotte TR. Adeno-associated virus-mediated gene transfer for lung diseases. Hum Gene Ther. 2005 Jun; 16(6):643-8.
Score: 0.150
-
Flotte TR. Recent developments in recombinant AAV-mediated gene therapy for lung diseases. Curr Gene Ther. 2005 Jun; 5(3):361-6.
Score: 0.150
-
Pires-Ferreira D, Reil D, Tang Q, Blackwood M, Gallagher T, Keeler AM, Chichester JA, Vyhnal KK, Lindborg JA, Benson J, Fu D, Flotte TR, Gruntman AM. Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy. Genes (Basel). 2024 Sep 10; 15(9).
Score: 0.143
-
Flotte TR. Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective. Hum Gene Ther. 2004 Jul; 15(7):716-7.
Score: 0.141
-
Conlon TJ, Flotte TR. Recombinant adeno-associated virus vectors for gene therapy. Expert Opin Biol Ther. 2004 Jul; 4(7):1093-101.
Score: 0.141
-
Flotte TR. Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2004 May; 11(10):805-10.
Score: 0.139
-
Daci R, Flotte TR. Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders. Int J Mol Sci. 2024 Jan 15; 25(2).
Score: 0.137
-
Gruntman AM, Xue W, Flotte TR. Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2024; 2750:11-17.
Score: 0.136
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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.
Score: 0.136
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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.
Score: 0.136
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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.
Score: 0.133
-
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.
Score: 0.131
-
Pires Ferreira D, Gruntman AM, Flotte TR. Gene therapy for alpha-1 antitrypsin deficiency: an update. Expert Opin Biol Ther. 2023 03; 23(3):283-291.
Score: 0.129
-
Arjomandnejad M, Dasgupta I, Flotte TR, Keeler AM. Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer. BioDrugs. 2023 May; 37(3):311-329.
Score: 0.129
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McDonald CL, Qasba P, Anderson DG, Bao G, Colvin RA, Kohn DB, Malik P, Mitchell MJ, Pu WT, Rawlings DJ, Williams DA, Flotte TR. Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Hum Gene Ther. 2023 02; 34(3-4):83-89.
Score: 0.128
-
Snyder RO, Flotte TR. Production of clinical-grade recombinant adeno-associated virus vectors. Curr Opin Biotechnol. 2002 Oct; 13(5):418-23.
Score: 0.125
-
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.
Score: 0.124
-
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.
Score: 0.124
-
Flotte TR. Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest. 2002 Mar; 121(3 Suppl):98S-102S.
Score: 0.120
-
Flotte TR, Gessler DJ. Gene Therapy for Rare Neurological Disorders. Clin Pharmacol Ther. 2022 04; 111(4):743-757.
Score: 0.120
-
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.
Score: 0.120
-
Flotte TR, Virella-Lowell I, Chesnut KA. Adeno-associated viral vectors for CF gene therapy. Methods Mol Med. 2002; 70:599-608.
Score: 0.119
-
Mah C, Byrne BJ, Flotte TR. Virus-based gene delivery systems. Clin Pharmacokinet. 2002; 41(12):901-11.
Score: 0.119
-
Flotte TR. Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Curr Opin Mol Ther. 2001 Oct; 3(5):497-502.
Score: 0.117
-
Flotte TR, Laube BL. Gene therapy in cystic fibrosis. Chest. 2001 Sep; 120(3 Suppl):124S-131S.
Score: 0.116
-
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.
Score: 0.116
-
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.
Score: 0.116
-
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.
Score: 0.115
-
Owen R, Flotte TR. Approaches and limitations to gene therapy for mitochondrial diseases. Antioxid Redox Signal. 2001 Jun; 3(3):451-60.
Score: 0.114
-
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.
Score: 0.113
-
Adair JE, Rubinger KS, Flotte TR. Women Lead Gene Therapy Science in 2021. Hum Gene Ther. 2021 04; 32(7-8):317-318.
Score: 0.113
-
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.
Score: 0.109
-
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.
Score: 0.109
-
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.
Score: 0.106
-
Flotte TR. Gene therapy for cystic fibrosis. Curr Opin Mol Ther. 1999 Aug; 1(4):510-6.
Score: 0.100
-
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.
Score: 0.099
-
Flotte TR. The Year in Review: The Top Five Papers of 2018. Hum Gene Ther. 2018 12; 29(12):1339-1340.
Score: 0.096
-
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.
Score: 0.093
-
Flotte TR. Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy. Hum Gene Ther. 2018 06; 29(6):641-642.
Score: 0.093
-
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.
Score: 0.092
-
Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol. 1998; 292:717-32.
Score: 0.090
-
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 a-1 Antitrypsin Deficiency. Mol Ther. 2017 11 01; 25(11):2477-2489.
Score: 0.088
-
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.
Score: 0.087
-
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.
Score: 0.086
-
Flotte TR, Ferkol TW. Genetic therapy. Past, present, and future. Pediatr Clin North Am. 1997 Feb; 44(1):153-78.
Score: 0.084
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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 a-1-antitrypsin deficiency. Proc Natl Acad Sci U S A. 2017 02 14; 114(7):1655-1659.
Score: 0.084
-
Flotte TR, Carter BJ. In vivo gene therapy with adeno-associated virus vectors for cystic fibrosis. Adv Pharmacol. 1997; 40:85-101.
Score: 0.084
-
Flotte TR. The Target's the Thing. Hum Gene Ther. 2016 07; 27(7):477.
Score: 0.081
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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.
Score: 0.081
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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.
Score: 0.080
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Carter BJ, Flotte TR. Development of adeno-associated virus vectors for gene therapy of cystic fibrosis. Curr Top Microbiol Immunol. 1996; 218:119-44.
Score: 0.078
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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.
Score: 0.078
-
Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy. Gene Ther. 1995 Aug; 2(6):357-62.
Score: 0.076
-
Afione SA, Conrad CK, Flotte TR. Gene therapy vectors as drug delivery systems. Clin Pharmacokinet. 1995 Mar; 28(3):181-9.
Score: 0.074
-
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.
Score: 0.073
-
Flotte TR. Prospects for virus-based gene therapy for cystic fibrosis. J Bioenerg Biomembr. 1993 Feb; 25(1):37-42.
Score: 0.064
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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, Lochm?ller 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.
Score: 0.064
-
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.
Score: 0.056
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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.
Score: 0.055
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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.
Score: 0.054
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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.
Score: 0.053
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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.
Score: 0.051
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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.
Score: 0.047
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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.
Score: 0.047
-
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.
Score: 0.045
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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, Yl?-Herttuala S. Progress and prospects: gene therapy clinical trials (part 1). Gene Ther. 2007 Oct; 14(20):1439-47.
Score: 0.044
-
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.
Score: 0.042
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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.
Score: 0.041
-
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.
Score: 0.041
-
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.
Score: 0.041
-
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.
Score: 0.038
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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.
Score: 0.037
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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.
Score: 0.036
-
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.
Score: 0.036
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Liang SQ, Navia AW, Ramseier M, Zhou X, Martinez M, Lee C, Zhou C, Wu J, Xie J, Su Q, Wang D, Flotte TR, Anderson DG, Tarantal AF, Shalek AK, Gao G, Xue W. AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys. Hum Gene Ther. 2024 10; 35(19-20):814-824.
Score: 0.035
-
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.
Score: 0.034
-
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.
Score: 0.033
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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.
Score: 0.032
-
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.
Score: 0.032
-
Ke H, Guay KP, Flotte TR, Gierasch LM, Gershenson A, Hebert DN. Secretion of functional a1-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.
Score: 0.031
-
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.
Score: 0.031
-
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.
Score: 0.031
-
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.
Score: 0.028
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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 Apr; 40(17):3060-3071.
Score: 0.028
-
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.
Score: 0.028
-
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.
Score: 0.026
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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.
Score: 0.024
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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.
Score: 0.023
-
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.
Score: 0.021
-
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.
Score: 0.020
-
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.
Score: 0.020
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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.
Score: 0.019
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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.
Score: 0.017
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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.
Score: 0.016
-
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.
Score: 0.016
-
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.
Score: 0.016
-
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.
Score: 0.011
-
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.
Score: 0.010
-
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.
Score: 0.010
-
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.
Score: 0.009
-
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.
Score: 0.009
-
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.
Score: 0.007