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263 related items for PubMed ID: 23756766
21. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. MacLaren RE, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Seymour L, Clark KR, During MJ, Cremers FP, Black GC, Lotery AJ, Downes SM, Webster AR, Seabra MC. Lancet; 2014 Mar 29; 383(9923):1129-37. PubMed ID: 24439297 [Abstract] [Full Text] [Related]
26. Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia: A Randomized Clinical Trial. Fischer MD, Ochakovski GA, Beier B, Seitz IP, Vaheb Y, Kortuem C, Reichel FFL, Kuehlewein L, Kahle NA, Peters T, Girach A, Zrenner E, Ueffing M, MacLaren RE, Bartz-Schmidt KU, Wilhelm B. JAMA Ophthalmol; 2019 Nov 01; 137(11):1247-1254. PubMed ID: 31465092 [Abstract] [Full Text] [Related]
36. Modeling Choroideremia Disease with Isogenic Induced Pluripotent Stem Cells. Fonseca AF, Coelho R, da-Silva ML, Lemos L, Hall MJ, Oliveira D, Falcão AS, Tenreiro S, Seabra MC, Antas P. Stem Cells Dev; 2024 Oct 01; 33(19-20):528-539. PubMed ID: 39078329 [Abstract] [Full Text] [Related]
37. Reduced Retinal Pigment Epithelial Autophagy Due to Loss of Rab12 Prenylation in a Human iPSC-RPE Model of Choroideremia. Raeker MÖ, Perera ND, Karoukis AJ, Chen L, Feathers KL, Ali RR, Thompson DA, Fahim AT. Cells; 2024 Jun 19; 13(12):. PubMed ID: 38920696 [Abstract] [Full Text] [Related]
38. Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina. Hickey DG, Edwards TL, Barnard AR, Singh MS, de Silva SR, McClements ME, Flannery JG, Hankins MW, MacLaren RE. Gene Ther; 2017 Dec 19; 24(12):787-800. PubMed ID: 28872643 [Abstract] [Full Text] [Related]