william hauswirth
william hauswirth
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Cited by
Gene therapy restores vision in a canine model of childhood blindness
GM Acland, GD Aguirre, J Ray, Q Zhang, TS Aleman, AV Cideciyan, ...
Nature genetics 28 (1), 92-95, 2001
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
A muscleblind knockout model for myotonic dystrophy
RN Kanadia, KA Johnstone, A Mankodi, C Lungu, CA Thornton, D Esson, ...
science 302 (5652), 1978-1980, 2003
A" humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells
S Zolotukhin, M Potter, WW Hauswirth, J Guy, N Muzyczka
Journal of virology 70 (7), 4646-4654, 1996
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
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
SG Jacobson, AV Cideciyan, R Ratnakaram, E Heon, SB Schwartz, ...
Archives of ophthalmology 130 (1), 9-24, 2012
DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration
H Kaneko, S Dridi, V Tarallo, BD Gelfand, BJ Fowler, WG Cho, ...
Nature 471 (7338), 325-330, 2011
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
DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88
V Tarallo, Y Hirano, BD Gelfand, S Dridi, N Kerur, Y Kim, WG Cho, ...
Cell 149 (4), 847-859, 2012
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
GM Acland, GD Aguirre, J Bennett, TS Aleman, AV Cideciyan, J Bennicelli, ...
Molecular Therapy 12 (6), 1072-1082, 2005
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement
AV Cideciyan, SG Jacobson, WA Beltran, A Sumaroka, M Swider, S Iwabe, ...
Proceedings of the National Academy of Sciences 110 (6), E517-E525, 2013
Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa
AS Lewin, KA Drenser, WW Hauswirth, S Nishikawa, D Yasumura, ...
Nature medicine 4 (8), 967-971, 1998
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus
JG Flannery, S Zolotukhin, MI Vaquero, MM LaVail, N Muzyczka, ...
Proceedings of the National Academy of Sciences 94 (13), 6916-6921, 1997
Mitochondrial DNA polymorphism in a maternal lineage of Holstein cows.
WW Hauswirth, PJ Laipis
Proceedings of the National Academy of Sciences 79 (15), 4686-4690, 1982
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
H Petrs-Silva, A Dinculescu, Q Li, SH Min, V Chiodo, JJ Pang, L Zhong, ...
Molecular therapy 17 (3), 463-471, 2009
Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model
KRG Martin, HA Quigley, DJ Zack, H Levkovitch-Verbin, J Kielczewski, ...
Investigative ophthalmology & visual science 44 (10), 4357-4365, 2003
Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year
AV Cideciyan, WW Hauswirth, TS Aleman, S Kaushal, SB Schwartz, ...
Human gene therapy 20 (9), 999-1004, 2009
Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells
AK Cheung, MD Hoggan, WW Hauswirth, KI Berns
Journal of virology 33 (2), 739-748, 1980
Improvement and decline in vision with gene therapy in childhood blindness
SG Jacobson, AV Cideciyan, AJ Roman, A Sumaroka, SB Schwartz, ...
New England Journal of Medicine 372 (20), 1920-1926, 2015
Gene therapy for red–green colour blindness in adult primates
K Mancuso, WW Hauswirth, Q Li, TB Connor, JA Kuchenbecker, ...
Nature 461 (7265), 784-787, 2009
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