Terence R Flotte
Terence R Flotte
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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
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
Adeno-associated virus vectors for gene therapy.
TR Flotte, BJ Carter
Gene therapy 2 (6), 357-362, 1995
Laboratory techniques in biochemistry and molecular biology
TR Flotte, KI Berns
Elsevier, 1969
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
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
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
Clinical gene therapy using recombinant adeno-associated virus vectors
C Mueller, TR Flotte
Gene therapy 15 (11), 858-863, 2008
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
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
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
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
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
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
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
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