{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T21:55:47Z","timestamp":1793656547378,"version":"4.5.1"},"reference-count":303,"publisher":"The Company of Biologists","issue":"2","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2015,2,1]]},"abstract":"<jats:p>Retinal neurodegeneration associated with the dysfunction or death of photoreceptors is a major cause of incurable vision loss. Tremendous progress has been made over the last two decades in discovering genes and genetic defects that lead to retinal diseases. The primary focus has now shifted to uncovering disease mechanisms and designing treatment strategies, especially inspired by the successful application of gene therapy in some forms of congenital blindness in humans. Both spontaneous and laboratory-generated mouse mutants have been valuable for providing fundamental insights into normal retinal development and for deciphering disease pathology. Here, we provide a review of mouse models of human retinal degeneration, with a primary focus on diseases affecting photoreceptor function. We also describe models associated with retinal pigment epithelium dysfunction or synaptic abnormalities. Furthermore, we highlight the crucial role of mouse models in elucidating retinal and photoreceptor biology in health and disease, and in the assessment of novel therapeutic modalities, including gene- and stem-cell-based therapies, for retinal degenerative diseases.<\/jats:p>","DOI":"10.1242\/dmm.017913","type":"journal-article","created":{"date-parts":[[2015,2,3]],"date-time":"2015-02-03T07:24:44Z","timestamp":1422948284000},"page":"109-129","source":"Crossref","is-referenced-by-count":235,"title":["Biology and therapy of inherited retinal degenerative disease: insights from mouse models"],"prefix":"10.1242","volume":"8","author":[{"given":"Shobi","family":"Veleri","sequence":"first","affiliation":[{"name":"Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Csilla H.","family":"Lazar","sequence":"additional","affiliation":[{"name":"Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA."},{"name":"Molecular Biology Center, Interdisciplinary Research Institute on Bio-Nano Sciences, Babes-Bolyai-University, Cluj-Napoca, 400271, Romania."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bo","family":"Chang","sequence":"additional","affiliation":[{"name":"The Jackson Laboratory, Bar Harbor, ME 04609, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul A.","family":"Sieving","sequence":"additional","affiliation":[{"name":"National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eyal","family":"Banin","sequence":"additional","affiliation":[{"name":"Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA."},{"name":"Center for Retinal and Macular Degenerations, Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem 91120, Israel."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anand","family":"Swaroop","sequence":"additional","affiliation":[{"name":"Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"237","reference":[{"key":"2021042501180641100_b1-0080109","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1038\/ng0501-92","article-title":"Gene therapy restores vision in a canine model of childhood blindness","volume":"28","author":"Acland","year":"2001","journal-title":"Nat. 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