{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T03:16:50Z","timestamp":1789183010002,"version":"build-2803163510"},"reference-count":75,"publisher":"MDPI AG","issue":"6","license":[{"start":{"date-parts":[[2024,5,28]],"date-time":"2024-05-28T00:00:00Z","timestamp":1716854400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Canada Graduate Scholarship (Canadian Institutes of Health Research) and the Vision Science Research Program Scholarship (University of Toronto and University Health Network)"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>Inherited retinal diseases (IRDs) are a large group of genetically and clinically diverse blinding eye conditions that result in progressive and irreversible photoreceptor degeneration and vision loss. To date, no cures have been found, although strides toward treatments for specific IRDs have been made in recent years. To accelerate treatment discovery, retinal organoids provide an ideal human IRD model. This review aims to give background on the development and importance of retinal organoids for the human-based in vitro study of the retina and human retinogenesis and retinal pathologies. From there, we explore retinal pathologies in the context of IRDs and the current landscape of IRD treatment discovery. We discuss the usefulness of retinal organoids in this context (as a patient-derived cell model for IRDs) to precisely understand the pathogenesis and potential mechanisms behind a specific IRD-causing variant of interest. Finally, we discuss the importance and promise of retinal organoids in treatment discovery for IRDs, now and in the future.<\/jats:p>","DOI":"10.3390\/genes15060705","type":"journal-article","created":{"date-parts":[[2024,5,28]],"date-time":"2024-05-28T05:19:49Z","timestamp":1716873589000},"page":"705","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Inherited Retinal Diseases and Retinal Organoids as Preclinical Cell Models for Inherited Retinal Disease Research"],"prefix":"10.3390","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4939-021X","authenticated-orcid":false,"given":"Kristen E.","family":"Ashworth","sequence":"first","affiliation":[{"name":"Institute of Medical Science, University of Toronto, Toronto, ON M5S 3H2, Canada"},{"name":"Donald K. Johnson Eye Institute, Toronto Western Hospital, Toronto, ON M5T 2S8, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Weisbrod","sequence":"additional","affiliation":[{"name":"Donald K. Johnson Eye Institute, Toronto Western Hospital, Toronto, ON M5T 2S8, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4644-3339","authenticated-orcid":false,"given":"Brian G.","family":"Ballios","sequence":"additional","affiliation":[{"name":"Institute of Medical Science, University of Toronto, Toronto, ON M5S 3H2, Canada"},{"name":"Donald K. Johnson Eye Institute, Toronto Western Hospital, Toronto, ON M5T 2S8, Canada"},{"name":"Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON M5T 3A9, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,5,28]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Martinez Velazquez, L.A., and Ballios, B.G. (2021). The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease. Int. J. Mol. Sci., 22.","DOI":"10.3390\/ijms222111542"},{"key":"ref_2","doi-asserted-by":"crossref","unstructured":"Dowling, J.E. (2012). The Retina: An Approachable Part of the Brain, Belknap Press of Harvard University Press. Rev. ed.","DOI":"10.2307\/j.ctv31zqj2d"},{"key":"ref_3","doi-asserted-by":"crossref","unstructured":"Quinn, P.M.J., and Wijnholds, J. (2019). Retinogenesis of the Human Fetal Retina: An Apical Polarity Perspective. 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