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10. Current Management of Patients with RPE65 Mutation-Associated Inherited Retinal Degenerations in Europe: Results of a Multinational Survey by the European Vision Institute Clinical Research Network. Lorenz B; Tavares J; van den Born LI; Marques JP; Scholl HPN; Ophthalmic Res; 2021; 64(5):740-753. PubMed ID: 33684911 [TBL] [Abstract][Full Text] [Related]
12. Epidemiology of Mutations in the 65-kDa Retinal Pigment Epithelium (RPE65) Gene-Mediated Inherited Retinal Dystrophies: A Systematic Literature Review. Sallum JMF; Kaur VP; Shaikh J; Banhazi J; Spera C; Aouadj C; Viriato D; Fischer MD Adv Ther; 2022 Mar; 39(3):1179-1198. PubMed ID: 35098484 [TBL] [Abstract][Full Text] [Related]
13. Available Evidence on Leber Congenital Amaurosis and Gene Therapy. Alkharashi M; Fulton AB Semin Ophthalmol; 2017; 32(1):14-21. PubMed ID: 27686653 [TBL] [Abstract][Full Text] [Related]
14. [Diagnostics of inherited retinal degenerations by gene therapy]. Shurygina MF; Khoteeva AM Vestn Oftalmol; 2021; 137(4):145-151. PubMed ID: 34410070 [TBL] [Abstract][Full Text] [Related]
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17. Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration. Cideciyan AV; Jacobson SG; Beltran WA; Hauswirth WW; Aguirre GD Proc Natl Acad Sci U S A; 2013 May; 110(19):E1706. PubMed ID: 23789127 [No Abstract] [Full Text] [Related]
18. [Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial]. Chacón-Camacho ÓF; Zenteno JC Gac Med Mex; 2017; 153(2):276-278. PubMed ID: 28474714 [TBL] [Abstract][Full Text] [Related]
19. Long-term effect of gene therapy on Leber's congenital amaurosis. Bainbridge JW; Mehat MS; Sundaram V; Robbie SJ; Barker SE; Ripamonti C; Georgiadis A; Mowat FM; Beattie SG; Gardner PJ; Feathers KL; Luong VA; Yzer S; Balaggan K; Viswanathan A; de Ravel TJ; Casteels I; Holder GE; Tyler N; Fitzke FW; Weleber RG; Nardini M; Moore AT; Thompson DA; Petersen-Jones SM; Michaelides M; van den Born LI; Stockman A; Smith AJ; Rubin G; Ali RR N Engl J Med; 2015 May; 372(20):1887-97. PubMed ID: 25938638 [TBL] [Abstract][Full Text] [Related]
20. Gene therapy for Leber congenital amaurosis: advances and future directions. Hufnagel RB; Ahmed ZM; Corrêa ZM; Sisk RA Graefes Arch Clin Exp Ophthalmol; 2012 Aug; 250(8):1117-28. PubMed ID: 22644094 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]