{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T16:22:21Z","timestamp":1792167741542,"version":"4.4.0"},"reference-count":61,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"7","content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2022,7]]},"abstract":"<jats:p>Inherited retinal diseases (IRD) are genotypically and phenotypically varied disorders that lead to progressive degeneration of the outer retina and the retinal pigment epithelium (RPE) eventually resulting in severe vision loss. Recent research and developments in gene therapy and cell therapy have shown therapeutic promise in these hitherto incurable diseases. In gene therapy, copies of a healthy gene are introduced into the host cells via a viral vector. Clinical trials for several genes are underway while treatment for RPE65 called voretigene neparvovec, is already approved and commercially available. Cell therapy involves the introduction of stem cells that can replace degenerated cells. These therapies are delivered to the target tissues, namely the photoreceptors (PR) and RPE via subretinal, intravitreal, or suprachoroidal delivery systems. Although there are several limitations to these therapies, they are expected to slow the disease progression and restore some visual functions. Further advances such as gene editing technologies are likely to result in more precise and personalized treatments. Currently, several IRDs such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, choroideremia, achromatopsia, and Usher syndrome are being evaluated for possible gene therapy or cell therapy. It is important to encourage patients to undergo gene testing and maintain a nationwide registry of IRDs. This article provides an overview of the basics of these therapies and their current status.<\/jats:p>","DOI":"10.4103\/ijo.ijo_82_22","type":"journal-article","created":{"date-parts":[[2022,7,12]],"date-time":"2022-07-12T06:28:32Z","timestamp":1657607312000},"page":"2316-2325","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":21,"title":["Newer therapeutic options for inherited retinal diseases: Gene and cell replacement therapy"],"prefix":"10.4103","volume":"70","author":[{"given":"Rajani","family":"Battu","sequence":"first","affiliation":[{"name":"Aster CMI Hospital, Bengaluru, Karnataka, India"},{"name":"Centre for Eye Genetics and Research, Bengaluru, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dhanashree","family":"Ratra","sequence":"additional","affiliation":[{"name":"Department of Vitreoretinal Diseases, Sankara Nethralaya, Chennai, Tamil Nadu, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lingam","family":"Gopal","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore Eye Research Institute, Singapore"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2022,6,30]]},"reference":[{"key":"B1-20240828","doi-asserted-by":"crossref","first-page":"757","DOI":"10.1007\/s00431-011-1615-2","article-title":"Retinal dystrophies and gene therapy","volume":"171","author":"Sundaram","year":"2012","journal-title":"Eur J Pediatr"},{"key":"B2-20240828","doi-asserted-by":"crossref","first-page":"5722","DOI":"10.3390\/ijms20225722","article-title":"Gene therapy in retinal dystrophies","volume":"20","author":"Ziccardi","year":"2019","journal-title":"Int J Mol Sci"},{"key":"B3-20240828","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1038\/gt.2008.66","article-title":"AAV-mediated gene therapy for retinal disorders:From mouse to man","volume":"15","author":"Buch","year":"2008","journal-title":"Gene Ther"},{"key":"B4-20240828","doi-asserted-by":"crossref","first-page":"4197","DOI":"10.3390\/ijms21124197","article-title":"Recombinant Adeno-Associated Viral Vectors (rAAV)-vector elements in ocular gene therapy clinical trials and transgene expression and bioactivity assays","volume":"21","author":"Buck","year":"2020","journal-title":"Int J Mol Sci"},{"key":"B5-20240828","doi-asserted-by":"crossref","first-page":"316","DOI":"10.3390\/v10060316","article-title":"Non-primate lentiviral vectors and their applications in gene therapy for ocular disorders","volume":"10","author":"Cavalieri","year":"2018","journal-title":"Viruses"},{"key":"B6-20240828","doi-asserted-by":"crossref","first-page":"1258096","DOI":"10.1126\/science.1258096","article-title":"Genome. 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