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Terence Flotte to Genetic Vectors

This is a "connection" page, showing publications Terence Flotte has written about Genetic Vectors.
Connection Strength

18.288
  1. Eichler F, Cataltepe OI, Daci R, Puri AS, Taghian T, Jiang X, Shazeeb MS, Kuhn A, Hader A, Celik H, Vardar Z, Lewis CJ, Artinian R, Nagy A, Vachha B, Thompson R, Gallagher T, Bateman S, Parzych J, Spanakis SG, Vaughn TA, Pier K, De Boever E, Abbott MA, D Ambrosio E, Kokoski D, Blackwood M, Drummond E, Ratai EM, Townsend EL, McLaughlin H, Tifft CJ, Keeler AM, Sena-Esteves M, Gray-Edwards HL, Flotte TR. Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial. Nat Med. 2025 Sep; 31(9):2927-2935.
    View in: PubMed
    Score: 0.625
  2. Flotte TR. Intrathecal gene therapy for neurologic disease in humans. Mol Ther. 2024 May 01; 32(5):1185-1186.
    View in: PubMed
    Score: 0.571
  3. Flotte TR, Gessler DJ. Gene Therapy for Rare Neurological Disorders. Clin Pharmacol Ther. 2022 04; 111(4):743-757.
    View in: PubMed
    Score: 0.491
  4. 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.
    View in: PubMed
    Score: 0.438
  5. Flotte TR. Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors. Hum Gene Ther. 2020 04; 31(7-8):398-399.
    View in: PubMed
    Score: 0.431
  6. 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.
    View in: PubMed
    Score: 0.411
  7. 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.
    View in: PubMed
    Score: 0.409
  8. Gruntman AM, Flotte TR. The rapidly evolving state of gene therapy. FASEB J. 2018 04; 32(4):1733-1740.
    View in: PubMed
    Score: 0.375
  9. 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.
    View in: PubMed
    Score: 0.371
  10. Flotte TR. One More Controversy: Adeno-Associated Virus in Stem Cells. Hum Gene Ther. 2017 06; 28(6):449.
    View in: PubMed
    Score: 0.354
  11. Keeler AM, ElMallah MK, Flotte TR. Gene Therapy 2017: Progress and Future Directions. Clin Transl Sci. 2017 07; 10(4):242-248.
    View in: PubMed
    Score: 0.353
  12. 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.
    View in: PubMed
    Score: 0.348
  13. 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.
    View in: PubMed
    Score: 0.324
  14. 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.
    View in: PubMed
    Score: 0.314
  15. 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.
    View in: PubMed
    Score: 0.305
  16. 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.
    View in: PubMed
    Score: 0.282
  17. 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.
    View in: PubMed
    Score: 0.277
  18. 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.
    View in: PubMed
    Score: 0.276
  19. 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.
    View in: PubMed
    Score: 0.246
  20. 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.
    View in: PubMed
    Score: 0.237
  21. Flotte TR, Conlon TJ, Mueller C. Preclinical study design for rAAV. Methods Mol Biol. 2011; 807:317-37.
    View in: PubMed
    Score: 0.227
  22. 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.
    View in: PubMed
    Score: 0.226
  23. Flotte TR. Sendai virus vectors. Pushing the envelope in the lung. Gene Ther. 2011 Feb; 18(2):107-8.
    View in: PubMed
    Score: 0.223
  24. 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.
    View in: PubMed
    Score: 0.195
  25. 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.
    View in: PubMed
    Score: 0.194
  26. 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.
    View in: PubMed
    Score: 0.191
  27. 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.
    View in: PubMed
    Score: 0.191
  28. Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 2008 Jun; 15(11):858-63.
    View in: PubMed
    Score: 0.188
  29. 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.
    View in: PubMed
    Score: 0.184
  30. Flotte TR. Gene therapy: the first two decades and the current state-of-the-art. J Cell Physiol. 2007 Nov; 213(2):301-5.
    View in: PubMed
    Score: 0.182
  31. 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.
    View in: PubMed
    Score: 0.174
  32. 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.
    View in: PubMed
    Score: 0.160
  33. Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res. 2005 Dec; 58(6):1143-7.
    View in: PubMed
    Score: 0.159
  34. 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.
    View in: PubMed
    Score: 0.155
  35. 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.
    View in: PubMed
    Score: 0.151
  36. 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.
    View in: PubMed
    Score: 0.148
  37. 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).
    View in: PubMed
    Score: 0.147
  38. 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.
    View in: PubMed
    Score: 0.146
  39. 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.
    View in: PubMed
    Score: 0.145
  40. Flotte TR. Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective. Hum Gene Ther. 2004 Jul; 15(7):716-7.
    View in: PubMed
    Score: 0.145
  41. Conlon TJ, Flotte TR. Recombinant adeno-associated virus vectors for gene therapy. Expert Opin Biol Ther. 2004 Jul; 4(7):1093-101.
    View in: PubMed
    Score: 0.145
  42. Flotte TR. Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2004 May; 11(10):805-10.
    View in: PubMed
    Score: 0.143
  43. 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.
    View in: PubMed
    Score: 0.140
  44. 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.
    View in: PubMed
    Score: 0.140
  45. 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.
    View in: PubMed
    Score: 0.135
  46. 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.
    View in: PubMed
    Score: 0.134
  47. 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.
    View in: PubMed
    Score: 0.132
  48. 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.
    View in: PubMed
    Score: 0.132
  49. Snyder RO, Flotte TR. Production of clinical-grade recombinant adeno-associated virus vectors. Curr Opin Biotechnol. 2002 Oct; 13(5):418-23.
    View in: PubMed
    Score: 0.128
  50. 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.
    View in: PubMed
    Score: 0.127
  51. 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.
    View in: PubMed
    Score: 0.127
  52. Flotte TR, Virella-Lowell I, Chesnut KA. Adeno-associated viral vectors for CF gene therapy. Methods Mol Med. 2002; 70:599-608.
    View in: PubMed
    Score: 0.122
  53. Mah C, Byrne BJ, Flotte TR. Virus-based gene delivery systems. Clin Pharmacokinet. 2002; 41(12):901-11.
    View in: PubMed
    Score: 0.122
  54. Flotte TR. Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Curr Opin Mol Ther. 2001 Oct; 3(5):497-502.
    View in: PubMed
    Score: 0.119
  55. 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.
    View in: PubMed
    Score: 0.119
  56. 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.
    View in: PubMed
    Score: 0.117
  57. 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.
    View in: PubMed
    Score: 0.115
  58. Flotte TR. Size does matter: overcoming the adeno-associated virus packaging limit. Respir Res. 2000; 1(1):16-8.
    View in: PubMed
    Score: 0.110
  59. Flotte TR. Barrie J. Carter: The Science and the Scientist. Hum Gene Ther. 2020 05; 31(9-10):489-490.
    View in: PubMed
    Score: 0.108
  60. 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.
    View in: PubMed
    Score: 0.106
  61. 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.
    View in: PubMed
    Score: 0.105
  62. Afione SA, Wang J, Walsh S, Guggino WB, Flotte TR. Delayed expression of adeno-associated virus vector DNA. Intervirology. 1999; 42(4):213-20.
    View in: PubMed
    Score: 0.099
  63. Flotte TR. Top Five Gene Therapy Stories of 2019. Hum Gene Ther. 2019 01; 30(1):1-2.
    View in: PubMed
    Score: 0.099
  64. Flotte TR. Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy. Hum Gene Ther. 2018 06; 29(6):641-642.
    View in: PubMed
    Score: 0.095
  65. 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.
    View in: PubMed
    Score: 0.094
  66. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol. 1998; 292:717-32.
    View in: PubMed
    Score: 0.092
  67. 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.
    View in: PubMed
    Score: 0.092
  68. 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.
    View in: PubMed
    Score: 0.090
  69. 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.
    View in: PubMed
    Score: 0.088
  70. Wilkins O, Keeler AM, Flotte TR. CAR T-Cell Therapy: Progress and Prospects. Hum Gene Ther Methods. 2017 04; 28(2):61-66.
    View in: PubMed
    Score: 0.087
  71. Flotte TR, Gao G. AAV Is Now a Medicine: We Had Better Get This Right. Hum Gene Ther. 2017 04; 28(4):307.
    View in: PubMed
    Score: 0.087
  72. Flotte TR. Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy? Hum Gene Ther. 2017 02; 28(2):147-148.
    View in: PubMed
    Score: 0.086
  73. Flotte TR, Carter BJ. In vivo gene therapy with adeno-associated virus vectors for cystic fibrosis. Adv Pharmacol. 1997; 40:85-101.
    View in: PubMed
    Score: 0.086
  74. 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.
    View in: PubMed
    Score: 0.084
  75. 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.
    View in: PubMed
    Score: 0.083
  76. 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.
    View in: PubMed
    Score: 0.082
  77. Carter BJ, Flotte TR. Development of adeno-associated virus vectors for gene therapy of cystic fibrosis. Curr Top Microbiol Immunol. 1996; 218:119-44.
    View in: PubMed
    Score: 0.080
  78. 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.
    View in: PubMed
    Score: 0.079
  79. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy. Gene Ther. 1995 Aug; 2(6):357-62.
    View in: PubMed
    Score: 0.078
  80. 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.
    View in: PubMed
    Score: 0.077
  81. Afione SA, Conrad CK, Flotte TR. Gene therapy vectors as drug delivery systems. Clin Pharmacokinet. 1995 Mar; 28(3):181-9.
    View in: PubMed
    Score: 0.076
  82. 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.
    View in: PubMed
    Score: 0.075
  83. Loring HS, Flotte TR. Current status of gene therapy for a-1 antitrypsin deficiency. Expert Opin Biol Ther. 2015 Mar; 15(3):329-36.
    View in: PubMed
    Score: 0.074
  84. 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.
    View in: PubMed
    Score: 0.074
  85. 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.
    View in: PubMed
    Score: 0.072
  86. 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.
    View in: PubMed
    Score: 0.067
  87. Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9.
    View in: PubMed
    Score: 0.066
  88. 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.
    View in: PubMed
    Score: 0.065
  89. 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.
    View in: PubMed
    Score: 0.064
  90. 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.
    View in: PubMed
    Score: 0.063
  91. 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.
    View in: PubMed
    Score: 0.062
  92. 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.
    View in: PubMed
    Score: 0.061
  93. 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.
    View in: PubMed
    Score: 0.057
  94. 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.
    View in: PubMed
    Score: 0.057
  95. 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, M?ller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivi?re 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.
    View in: PubMed
    Score: 0.056
  96. 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.
    View in: PubMed
    Score: 0.052
  97. 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.
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    Score: 0.052
  98. 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.
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    Score: 0.052
  99. Mueller C, Flotte TR. Gene therapy for cystic fibrosis. Clin Rev Allergy Immunol. 2008 Dec; 35(3):164-78.
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    Score: 0.049
  100. 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.
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    Score: 0.049
  101. 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.
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    Score: 0.047
  102. Flotte TR. In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negative. Mol Ther. 2008 May; 16(5):806-7.
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    Score: 0.047
  103. 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.
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    Score: 0.046
  104. 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.
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    Score: 0.045
  105. 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.
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    Score: 0.044
  106. 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.
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    Score: 0.044
  107. 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.
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    Score: 0.044
  108. 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.
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    Score: 0.043
  109. 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.
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    Score: 0.043
  110. 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.
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    Score: 0.043
  111. 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.
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    Score: 0.041
  112. Xing R, Xu M, Reil D, Destefano A, Cui M, Liu N, Liang J, Xu G, Luo L, Xu M, Zhang F, Tai PWL, Wei Y, Gruntman AM, Flotte TR, Gao G, Wang D. A single amino acid variant in the variable region I of AAV capsid confers liver detargeting. PLoS Pathog. 2025 Sep; 21(9):e1013533.
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    Score: 0.039
  113. 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.
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    Score: 0.039
  114. 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.
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    Score: 0.039
  115. 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.
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    Score: 0.039
  116. Flotte TR. Adeno-associated virus-mediated gene transfer for lung diseases. Hum Gene Ther. 2005 Jun; 16(6):643-8.
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    Score: 0.039
  117. 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.
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    Score: 0.038
  118. 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.
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    Score: 0.038
  119. 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.
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    Score: 0.035
  120. 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.
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    Score: 0.035
  121. 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.
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    Score: 0.034
  122. Flotte TR. The signal and the trap: targeted delivery and retention of proteins in the mitochondrion. Mol Ther. 2003 Jun; 7(6):715-6.
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    Score: 0.034
  123. 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.
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    Score: 0.033
  124. 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.
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    Score: 0.032
  125. 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.
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    Score: 0.031
  126. Flotte TR. Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest. 2002 Mar; 121(3 Suppl):98S-102S.
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    Score: 0.031
  127. 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.
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    Score: 0.031
  128. 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.
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    Score: 0.030
  129. Flotte TR, Laube BL. Gene therapy in cystic fibrosis. Chest. 2001 Sep; 120(3 Suppl):124S-131S.
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    Score: 0.030
  130. 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.
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    Score: 0.030
  131. 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.
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    Score: 0.029
  132. 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.
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    Score: 0.029
  133. 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.
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    Score: 0.028
  134. 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.
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    Score: 0.028
  135. 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.
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    Score: 0.027
  136. 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.
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    Score: 0.026
  137. Flotte TR. Gene therapy for cystic fibrosis. Curr Opin Mol Ther. 1999 Aug; 1(4):510-6.
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    Score: 0.026
  138. 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.
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    Score: 0.025
  139. 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.
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    Score: 0.025
  140. 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.
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    Score: 0.025
  141. 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.
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    Score: 0.024
  142. 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.
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    Score: 0.024
  143. 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.
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    Score: 0.023
  144. 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.
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    Score: 0.023
  145. Flotte TR, Ferkol TW. Genetic therapy. Past, present, and future. Pediatr Clin North Am. 1997 Feb; 44(1):153-78.
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    Score: 0.022
  146. 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.
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    Score: 0.021
  147. 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.
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    Score: 0.020
  148. 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.
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    Score: 0.020
  149. 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.
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    Score: 0.020
  150. 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.
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    Score: 0.017
  151. 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.
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    Score: 0.017
  152. 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.
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    Score: 0.016
  153. Flotte TR. Prospects for virus-based gene therapy for cystic fibrosis. J Bioenerg Biomembr. 1993 Feb; 25(1):37-42.
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    Score: 0.016
  154. 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.
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    Score: 0.013
  155. 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.
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    Score: 0.010
  156. 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.
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    Score: 0.010
  157. 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.
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    Score: 0.009
  158. 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.
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  159. 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.
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    Score: 0.008
  160. 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.
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    Score: 0.008
  161. 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.
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    Score: 0.008
  162. 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.
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  163. 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.
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    Score: 0.007
  164. 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.
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    Score: 0.007
  165. 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.
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    Score: 0.006
  166. 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.
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    Score: 0.006
  167. 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.
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    Score: 0.005
  168. 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.
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