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9. Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Testa F; Maguire AM; Rossi S; Pierce EA; Melillo P; Marshall K; Banfi S; Surace EM; Sun J; Acerra C; Wright JF; Wellman J; High KA; Auricchio A; Bennett J; Simonelli F Ophthalmology; 2013 Jun; 120(6):1283-91. PubMed ID: 23474247 [TBL] [Abstract][Full Text] [Related]
10. 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]
11. 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]
12. In utero gene therapy rescues vision in a murine model of congenital blindness. Dejneka NS; Surace EM; Aleman TS; Cideciyan AV; Lyubarsky A; Savchenko A; Redmond TM; Tang W; Wei Z; Rex TS; Glover E; Maguire AM; Pugh EN; Jacobson SG; Bennett J Mol Ther; 2004 Feb; 9(2):182-8. PubMed ID: 14759802 [TBL] [Abstract][Full Text] [Related]
13. 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]
14. 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]
15. Safety and efficacy of gene transfer for Leber's congenital amaurosis. Maguire AM; Simonelli F; Pierce EA; Pugh EN; Mingozzi F; Bennicelli J; Banfi S; Marshall KA; Testa F; Surace EM; Rossi S; Lyubarsky A; Arruda VR; Konkle B; Stone E; Sun J; Jacobs J; Dell'Osso L; Hertle R; Ma JX; Redmond TM; Zhu X; Hauck B; Zelenaia O; Shindler KS; Maguire MG; Wright JF; Volpe NJ; McDonnell JW; Auricchio A; High KA; Bennett J N Engl J Med; 2008 May; 358(21):2240-8. PubMed ID: 18441370 [TBL] [Abstract][Full Text] [Related]
16. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye. Li W; Kong F; Li X; Dai X; Liu X; Zheng Q; Wu R; Zhou X; Lü F; Chang B; Li Q; Hauswirth WW; Qu J; Pang JJ Mol Vis; 2009; 15():267-75. PubMed ID: 19190735 [TBL] [Abstract][Full Text] [Related]
17. Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. Pennesi ME; Weleber RG; Yang P; Whitebirch C; Thean B; Flotte TR; Humphries M; Chegarnov E; Beasley KN; Stout JT; Chulay JD Hum Gene Ther; 2018 Dec; 29(12):1428-1437. PubMed ID: 29869534 [TBL] [Abstract][Full Text] [Related]
18. Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice. Barker SE; Broderick CA; Robbie SJ; Duran Y; Natkunarajah M; Buch P; Balaggan KS; MacLaren RE; Bainbridge JW; Smith AJ; Ali RR J Gene Med; 2009 Jun; 11(6):486-97. PubMed ID: 19340848 [TBL] [Abstract][Full Text] [Related]
19. The human visual cortex responds to gene therapy-mediated recovery of retinal function. Ashtari M; Cyckowski LL; Monroe JF; Marshall KA; Chung DC; Auricchio A; Simonelli F; Leroy BP; Maguire AM; Shindler KS; Bennett J J Clin Invest; 2011 Jun; 121(6):2160-8. PubMed ID: 21606598 [TBL] [Abstract][Full Text] [Related]
20. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. Weleber RG; Pennesi ME; Wilson DJ; Kaushal S; Erker LR; Jensen L; McBride MT; Flotte TR; Humphries M; Calcedo R; Hauswirth WW; Chulay JD; Stout JT Ophthalmology; 2016 Jul; 123(7):1606-20. PubMed ID: 27102010 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]