These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
4. 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. Hauswirth WW; Aleman TS; Kaushal S; Cideciyan AV; Schwartz SB; Wang L; Conlon TJ; Boye SL; Flotte TR; Byrne BJ; Jacobson SG Hum Gene Ther; 2008 Oct; 19(10):979-90. PubMed ID: 18774912 [TBL] [Abstract][Full Text] [Related]
5. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Simonelli F; Maguire AM; Testa F; Pierce EA; Mingozzi F; Bennicelli JL; Rossi S; Marshall K; Banfi S; Surace EM; Sun J; Redmond TM; Zhu X; Shindler KS; Ying GS; Ziviello C; Acerra C; Wright JF; McDonnell JW; High KA; Bennett J; Auricchio A Mol Ther; 2010 Mar; 18(3):643-50. PubMed ID: 19953081 [TBL] [Abstract][Full Text] [Related]
6. 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. Acland GM; Aguirre GD; Bennett J; Aleman TS; Cideciyan AV; Bennicelli J; Dejneka NS; Pearce-Kelling SE; Maguire AM; Palczewski K; Hauswirth WW; Jacobson SG Mol Ther; 2005 Dec; 12(6):1072-82. PubMed ID: 16226919 [TBL] [Abstract][Full Text] [Related]
7. Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness. Amado D; Mingozzi F; Hui D; Bennicelli JL; Wei Z; Chen Y; Bote E; Grant RL; Golden JA; Narfstrom K; Syed NA; Orlin SE; High KA; Maguire AM; Bennett J Sci Transl Med; 2010 Mar; 2(21):21ra16. PubMed ID: 20374996 [TBL] [Abstract][Full Text] [Related]
8. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Bennett J; Wellman J; Marshall KA; McCague S; Ashtari M; DiStefano-Pappas J; Elci OU; Chung DC; Sun J; Wright JF; Cross DR; Aravand P; Cyckowski LL; Bennicelli JL; Mingozzi F; Auricchio A; Pierce EA; Ruggiero J; Leroy BP; Simonelli F; High KA; Maguire AM Lancet; 2016 Aug; 388(10045):661-72. PubMed ID: 27375040 [TBL] [Abstract][Full Text] [Related]
9. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Jacobson SG; Boye SL; Aleman TS; Conlon TJ; Zeiss CJ; Roman AJ; Cideciyan AV; Schwartz SB; Komaromy AM; Doobrajh M; Cheung AY; Sumaroka A; Pearce-Kelling SE; Aguirre GD; Kaushal S; Maguire AM; Flotte TR; Hauswirth WW Hum Gene Ther; 2006 Aug; 17(8):845-58. PubMed ID: 16942444 [TBL] [Abstract][Full Text] [Related]
10. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Jacobson SG; Cideciyan AV; Ratnakaram R; Heon E; Schwartz SB; Roman AJ; Peden MC; Aleman TS; Boye SL; Sumaroka A; Conlon TJ; Calcedo R; Pang JJ; Erger KE; Olivares MB; Mullins CL; Swider M; Kaushal S; Feuer WJ; Iannaccone A; Fishman GA; Stone EM; Byrne BJ; Hauswirth WW Arch Ophthalmol; 2012 Jan; 130(1):9-24. PubMed ID: 21911650 [TBL] [Abstract][Full Text] [Related]
11. RPE65: role in the visual cycle, human retinal disease, and gene therapy. Cai X; Conley SM; Naash MI Ophthalmic Genet; 2009 Jun; 30(2):57-62. PubMed ID: 19373675 [TBL] [Abstract][Full Text] [Related]