{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T14:11:45Z","timestamp":1790345505939,"version":"4.1.0"},"reference-count":45,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"51","content-domain":{"domain":["www.science.org"],"crossmark-restriction":true},"short-container-title":["Sci. Adv."],"published-print":{"date-parts":[[2024,12,20]]},"abstract":"<jats:p>\n                    Retinitis pigmentosa (RP) is characterized by degeneration of rod and cone photoreceptors that progresses to irreversible blindness. Now, there are no mutation-agnostic approaches to treat RP. Here, we utilized a single adeno-associated virus (AAV)\u2013based CRISPR activation system to activate phosphodiesterase 6B (Pde6b) to mitigate the severe degeneration in\n                    <jats:italic>Pde6a<\/jats:italic>\n                    <jats:sup>nmf363<\/jats:sup>\n                    mice. We demonstrate that transcriptional activation of\n                    <jats:italic>Pde6b<\/jats:italic>\n                    can rescue the loss of\n                    <jats:italic>Pde6a<\/jats:italic>\n                    , with preservation of retinal structure, restoration of electroretinography responses, and improvement of visual function as assessed by optokinetic response and looming-induced escape behaviors. These findings demonstrate the therapeutic potential of a dCasMINI-mediated activation strategy that provides a mutation-independent treatment for retinal degeneration. This study offers a promising therapeutic approach for RP and potentially other forms of genetic diseases.\n                  <\/jats:p>","DOI":"10.1126\/sciadv.adn7540","type":"journal-article","created":{"date-parts":[[2024,12,18]],"date-time":"2024-12-18T13:58:47Z","timestamp":1734530327000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":9,"title":["dCasMINI-mediated therapy rescues photoreceptors degeneration in a mouse model of retinitis pigmentosa"],"prefix":"10.1126","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-8194-5680","authenticated-orcid":true,"given":"Qing","family":"Wang","sequence":"first","affiliation":[{"name":"Byers Eye Institute, Department of Ophthalmology, Stanford University, Palo Alto, CA 94304, 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