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J. Med."},{"key":"10.1016\/j.ymthe.2024.10.017_bib108","first-page":"1195","article-title":"Retinal gene therapy for choroideremia in a multicenter dose escalation phase I\/II clinical trial","volume":"59","author":"MacLaren","year":"2018","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.017_bib109","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1097\/IAE.0000000000002360","article-title":"Changes in Retinal Sensitivity after Gene Therapy in Choroideremia","volume":"40","author":"Fischer","year":"2020","journal-title":"Retina"},{"key":"10.1016\/j.ymthe.2024.10.017_bib110","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.ajo.2018.06.011","article-title":"Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience","volume":"193","author":"Dimopoulos","year":"2018","journal-title":"Am. J. 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Med."},{"key":"10.1016\/j.ymthe.2024.10.017_bib114","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/j.preteyeres.2018.03.005","article-title":"Non-syndromic retinitis pigmentosa","volume":"66","author":"Verbakel","year":"2018","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.ymthe.2024.10.017_bib115","doi-asserted-by":"crossref","first-page":"462","DOI":"10.1038\/78182","article-title":"Mutational hot spot within a new RPGR exon in X-linked retinitis pigmentosa","volume":"25","author":"Vervoort","year":"2000","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2024.10.017_bib116","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1016\/j.exer.2015.06.007","article-title":"RPGR: Its role in photoreceptor physiology, human disease, and future therapies","volume":"138","author":"Megaw","year":"2015","journal-title":"Exp. 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Ther."},{"key":"10.1016\/j.ymthe.2024.10.017_bib119","doi-asserted-by":"crossref","first-page":"4558","DOI":"10.1167\/iovs.18-24931","article-title":"Progression in X-linked Retinitis Pigmentosa Due to ORF15-RPGR Mutations: Assessment of Localized Vision Changes Over 2 Years","volume":"59","author":"Cideciyan","year":"2018","journal-title":"Invest. Ophthalmol. Vis. 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