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The eye\u2019s small, compartmentalized structure, relative immune privilege, and direct accessibility through subretinal or intravitreal routes make it an ideal target for localized delivery with minimal systemic exposure. The approval of voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy marked a pivotal milestone, establishing proof of concept for durable and safe gene replacement therapy. Looking ahead, continued refinements in vector design, CRISPR-based editing strategies, and delivery platforms are expected to expand the therapeutic reach of gene therapy beyond monogenic disorders. With multiple early-phase clinical trials underway for inherited and acquired retinal diseases, the coming decade is poised to bring broader applicability, improved durability, and more accessible gene-based treatments across the spectrum of retinal pathology.<\/jats:p>","DOI":"10.3390\/jpm15120619","type":"journal-article","created":{"date-parts":[[2025,12,11]],"date-time":"2025-12-11T11:17:27Z","timestamp":1765451847000},"page":"619","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Gene Therapy for Inherited Retinal Disease: Current Strategies, Personalized Medicine, and Future Implications\u2014A Comprehensive Review"],"prefix":"10.3390","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0009-0004-1025-4765","authenticated-orcid":false,"given":"Fahad R.","family":"Butt","sequence":"first","affiliation":[{"name":"Schulich School of Medicine & Dentistry, University of Western Ontario, London, ON N6A 5B8, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0571-2894","authenticated-orcid":false,"given":"Thanansayan","family":"Dhivagaran","sequence":"additional","affiliation":[{"name":"Schulich School of Medicine & Dentistry, University of Western Ontario, London, ON N6A 5B8, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6834-9466","authenticated-orcid":false,"given":"Boaz","family":"Li","sequence":"additional","affiliation":[{"name":"Faculty of Medicine, The University of British Columbia, Vancouver, BC V6T 1Z4, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-8427-5484","authenticated-orcid":false,"given":"Mark","family":"Ashamalla","sequence":"additional","affiliation":[{"name":"Faculty of Medicine, The University of British Columbia, Vancouver, BC V6T 1Z4, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7069-3162","authenticated-orcid":false,"given":"Brendan K.","family":"Tao","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology & Vision Sciences, University of Toronto, Toronto, ON M5S 1A1, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Balas","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology & Vision Sciences, University of Toronto, Toronto, ON M5S 1A1, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Austin","family":"Pereira","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology & Vision Sciences, University of Toronto, Toronto, ON M5S 1A1, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peng","family":"Yan","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology & Vision Sciences, University of Toronto, Toronto, ON M5S 1A1, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parnian","family":"Arjmand","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology & Vision Sciences, University of Toronto, Toronto, ON M5S 1A1, Canada"},{"name":"School of Medicine, Toronto Metropolitan University, Toronto, ON M5B 2K3, Canada"},{"name":"Mississauga Retina Institute, 1420 Burnhamthorpe Rd. 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