|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
|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
|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
|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
|Adeno-associated virus vectors for gene therapy.|
TR Flotte, BJ Carter
Gene therapy 2 (6), 357, 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
|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
|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
|Clinical gene therapy using recombinant adeno-associated virus vectors|
C Mueller, TR Flotte
Gene therapy 15 (11), 858-863, 2008
|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
|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
|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
|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
|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
|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
|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
|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
|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
|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
|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 …|
TR Flotte, PL Zeitlin, TC Reynolds, AE Heald, P Pedersen, S Beck, ...
Human gene therapy 14 (11), 1079-1088, 2003