| 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 WW Hauswirth, TS Aleman, S Kaushal, AV Cideciyan, SB Schwartz, ... Human gene therapy 19 (10), 979-990, 2008 | 1144 | 2008 |
| Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics AV Cideciyan, TS Aleman, SL Boye, SB Schwartz, S Kaushal, AJ Roman, ... Proceedings of the National Academy of Sciences 105 (39), 15112-15117, 2008 | 784 | 2008 |
| Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector TR Flotte, SA Afione, C Conrad, SA McGrath, R Solow, H Oka, PL Zeitlin, ... Proceedings of the National Academy of Sciences 90 (22), 10613-10617, 1993 | 713 | 1993 |
| Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors S Zolotukhin, M Potter, I Zolotukhin, Y Sakai, S Loiler, TJ Fraites Jr, ... Methods 28 (2), 158-167, 2002 | 650 | 2002 |
| Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism P Wu, W Xiao, T Conlon, J Hughes, M Agbandje-McKenna, T Ferkol, ... Journal of virology 74 (18), 8635-8647, 2000 | 548 | 2000 |
| Adeno-associated virus vectors for gene therapy. TR Flotte, BJ Carter Gene therapy 2 (6), 357-362, 1995 | 529 | 1995 |
| Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR M Egan, T Flotte, S Afione, R Solow, PL Zeitlin, BJ Carter, WB Guggino Nature 358 (6387), 581-584, 1992 | 516 | 1992 |
| Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. TR Flotte, SA Afione, R Solow, ML Drumm, D Markakis, WB Guggino, ... Journal of Biological Chemistry 268 (5), 3781-3790, 1993 | 479 | 1993 |
| Results at 2 years after gene therapy for RPE65-deficient Leber congenital amaurosis and severe early-childhood–onset retinal dystrophy RG Weleber, ME Pennesi, DJ Wilson, S Kaushal, LR Erker, L Jensen, ... Ophthalmology 123 (7), 1606-1620, 2016 | 405* | 2016 |
| Clinical gene therapy using recombinant adeno-associated virus vectors C Mueller, TR Flotte Gene therapy 15 (11), 858-863, 2008 | 400 | 2008 |
| Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy ML Brantly, JD Chulay, L Wang, C Mueller, M Humphries, LT Spencer, ... Proceedings of the National Academy of Sciences 106 (38), 16363-16368, 2009 | 370 | 2009 |
| Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results TR Flotte, BC Trapnell, M Humphries, B Carey, R Calcedo, F Rouhani, ... Human gene therapy 22 (10), 1239-1247, 2011 | 369 | 2011 |
| A phase i study of an adeno-associated virus-cftr gene vector in adult cf patients with mild lung disease. Johns hopkins children's center, baltimore, maryland T Flotte, B Carter, C Conrad, W Guggino, T Reynolds, B Rosenstein, ... Human gene therapy 7 (9), 1145-1159, 1996 | 356 | 1996 |
| Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors S Song, M Morgan, T Ellis, A Poirier, K Chesnut, J Wang, M Brantly, ... Proceedings of the National Academy of Sciences 95 (24), 14384-14388, 1998 | 353 | 1998 |
| In vivo model of adeno-associated virus vector persistence and rescue SA Afione, CK Conrad, WG Kearns, S Chunduru, R Adams, TC Reynolds, ... Journal of virology 70 (5), 3235-3241, 1996 | 338 | 1996 |
| α-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis I Petrache, I Fijalkowska, TR Medler, J Skirball, P Cruz, L Zhen, ... The American journal of pathology 169 (4), 1155-1166, 2006 | 330 | 2006 |
| Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors A Donsante, C Vogler, N Muzyczka, JM Crawford, J Barker, T Flotte, ... Gene therapy 8 (17), 1343-1346, 2001 | 320 | 2001 |
| Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. TR Flotte, SA Afione, PL Zeitlin American journal of respiratory cell and molecular biology 11 (5), 517-521, 1994 | 316 | 1994 |
| A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies JA Wagner, IB Nepomuceno, AH Messner, ML Moran, EP Batson, ... Human gene therapy 13 (11), 1349-1359, 2002 | 306 | 2002 |
| Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. WG Kearns, SA Afione, SB Fulmer, MC Pang, D Erikson, M Egan, ... Gene Therapy 3 (9), 748-755, 1996 | 279 | 1996 |
Christian MuellerGlobal Head of Genomic Medicine -Sanofi GenzymeVerified email at sanofi.com
