| 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 | 1550 | 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 | 1207 | 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 | 885 | 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 | 839 | 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 | 825 | 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 | 790 | 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 | 717 | 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 | 678 | 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 | 676 | 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 | 579 | 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 | 538 | 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 | 525 | 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 | 515 | 1997 |
| 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 | 498 | 2009 |
| 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 | 496 | 1982 |
| 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 | 493 | 2003 |
| 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 | 434 | 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 | 429 | 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 | 428 | 2009 |
| 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 | 421 | 2009 |
Sanford L. BoyePowell Gene Therapy Center, Department of Pediatrics, University of FloridaPreverjeni e-poštni naslov na ufl.edu
