{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T01:55:09Z","timestamp":1773366909724,"version":"3.50.1"},"reference-count":178,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"funder":[{"DOI":"10.13039\/100007337","name":"Mass Eye and Ear","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007337","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Aspects of Medicine"],"published-print":{"date-parts":[[2026,4]]},"DOI":"10.1016\/j.mam.2026.101462","type":"journal-article","created":{"date-parts":[[2026,2,16]],"date-time":"2026-02-16T04:37:16Z","timestamp":1771216636000},"page":"101462","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"special_numbering":"C","title":["CRISPR as a therapeutic tool for inherited retinal degenerations: Advances, challenges, and future directions"],"prefix":"10.1016","volume":"108","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1323-8806","authenticated-orcid":false,"given":"Egle","family":"Galdikaite-Braziene","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raulas","family":"Kru\u0161nauskas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emiline","family":"Henderson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kinga M.","family":"Bujakowska","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.mam.2026.101462_bib1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1089\/nat.2016.0657","article-title":"FDA approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen Saga","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.mam.2026.101462_bib2","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1038\/nature24049","article-title":"RNA targeting with CRISPR-Cas13a","volume":"550","author":"Abudayyeh","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib3","doi-asserted-by":"crossref","DOI":"10.1126\/science.aaf5573","article-title":"C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector","volume":"353","author":"Abudayyeh","year":"2016","journal-title":"Science (New York, N.Y.)"},{"key":"10.1016\/j.mam.2026.101462_bib4","doi-asserted-by":"crossref","first-page":"817","DOI":"10.1016\/j.cell.2015.03.023","article-title":"Rod-derived cone viability factor promotes cone survival by stimulating aerobic glycolysis","volume":"161","author":"A\u00eft-Ali","year":"2015","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib5","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib6","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib7","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1016\/j.cell.2020.05.037","article-title":"Determinants of base editing outcomes from target library analysis and machine learning","volume":"182","author":"Arbab","year":"2020","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib8","doi-asserted-by":"crossref","first-page":"556","DOI":"10.1038\/mt.2015.220","article-title":"In vivo CRISPR\/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa","volume":"24","author":"Bakondi","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib9","doi-asserted-by":"crossref","first-page":"1709","DOI":"10.1126\/science.1138140","article-title":"CRISPR provides acquired resistance against viruses in prokaryotes","volume":"315","author":"Barrangou","year":"2007","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib10","doi-asserted-by":"crossref","DOI":"10.1016\/j.ymthe.2025.04.027","article-title":"Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library","author":"Becker","year":"2025","journal-title":"Mol. Ther."},{"issue":"1 10","key":"10.1016\/j.mam.2026.101462_bib11","first-page":"1","article-title":"Restoration of high-sensitivity and adapting vision with a cone opsin","volume":"10","author":"Berry","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib12","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1016\/S0959-437X(00)00195-7","article-title":"Molecular genetics and prospects for therapy of the inherited retinal dystrophies","volume":"11","author":"Bessant","year":"2001","journal-title":"Curr. Opin. Genet. Dev."},{"key":"10.1016\/j.mam.2026.101462_bib13","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.ajo.2013.03.021","article-title":"Randomized trial of ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for retinitis pigmentosa","volume":"156","author":"Birch","year":"2013","journal-title":"Am. J. Ophthalmol."},{"key":"10.1016\/j.mam.2026.101462_bib14","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1126\/science.270.5235.475","article-title":"T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years","volume":"270","author":"Blaese","year":"1995","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib15","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aba5614","article-title":"A gene therapy for inherited blindness using dCas9-VPR-mediated transcriptional activation","volume":"6","author":"B\u00f6hm","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.mam.2026.101462_bib16","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.12242","article-title":"Rhodopsin targeted transcriptional silencing by DNA-binding","volume":"5","author":"Botta","year":"2016","journal-title":"eLife"},{"key":"10.1016\/j.mam.2026.101462_bib17","first-page":"1","article-title":"Discovery and engineering of retrons for precise genome editing","author":"Buffington","year":"2025","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib18","doi-asserted-by":"crossref","first-page":"1999","DOI":"10.1016\/j.ymthe.2017.05.015","article-title":"Using CRISPR-Cas9 to generate gene-corrected autologous iPSCs for the treatment of inherited retinal degeneration","volume":"25","author":"Burnight","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib19","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aav3335","article-title":"In vivo genome editing rescues photoreceptor degeneration via a Cas9\/RecA-mediated homology-directed repair pathway","volume":"5","author":"Cai","year":"2019","journal-title":"Sci. Adv."},{"key":"10.1016\/j.mam.2026.101462_bib20","article-title":"Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases","volume":"2014","author":"Caia","year":"2014","journal-title":"eLife"},{"key":"10.1016\/j.mam.2026.101462_bib21","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1038\/s41591-020-0763-1","article-title":"Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR","volume":"26","author":"Cehajic-Kapetanovic","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.mam.2026.101462_bib22","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1038\/s41591-018-0326-x","article-title":"Identification of preexisting adaptive immunity to Cas9 proteins in humans","volume":"25","author":"Charlesworth","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.mam.2026.101462_bib23","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1038\/nmeth.3312","article-title":"Highly efficient Cas9-mediated transcriptional programming","volume":"12","author":"Chavez","year":"2015","journal-title":"Nat. Methods"},{"key":"10.1016\/j.mam.2026.101462_bib24","doi-asserted-by":"crossref","DOI":"10.1186\/s13287-023-03526-x","article-title":"Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis","volume":"14","author":"Chen","year":"2023","journal-title":"Stem Cell Res. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib25","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1038\/cr.2013.122","article-title":"Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system","volume":"23","author":"Cheng","year":"2013","journal-title":"Cell Res."},{"key":"10.1016\/j.mam.2026.101462_bib26","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.25946","article-title":"Glycolytic reliance promotes anabolism in photoreceptors","volume":"6","author":"Chinchore","year":"2017","journal-title":"eLife"},{"key":"10.1016\/j.mam.2026.101462_bib27","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-022-29490-3","article-title":"In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration","volume":"13","author":"Choi","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib28","doi-asserted-by":"crossref","first-page":"E8547","DOI":"10.1073\/pnas.1805055115","article-title":"Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector","volume":"115","author":"Cideciyan","year":"2018","journal-title":"Proc. Natl. Acad. Sci. U. S. A"},{"key":"10.1016\/j.mam.2026.101462_bib29","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"Cong","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib30","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1126\/science.aaq0180","article-title":"RNA editing with CRISPR-Cas13","volume":"358","author":"Cox","year":"2017","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib31","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-018-05843-9","article-title":"Cas9 immunity creates challenges for CRISPR gene editing therapies","volume":"9","author":"Crudele","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib32","doi-asserted-by":"crossref","first-page":"1338","DOI":"10.3390\/biology11091338","article-title":"Drug discovery strategies for inherited retinal degenerations","volume":"11","author":"Das","year":"2022","journal-title":"Biology"},{"key":"10.1016\/j.mam.2026.101462_bib33","doi-asserted-by":"crossref","first-page":"4623","DOI":"10.1113\/JP282076","article-title":"Optogenetic approaches to therapy for inherited retinal degenerations","volume":"600","author":"De","year":"2022","journal-title":"J. Physiol."},{"key":"10.1016\/j.mam.2026.101462_bib34","doi-asserted-by":"crossref","first-page":"556","DOI":"10.1086\/507318","article-title":"Mutations in the CEP290 (NPHP6) gene are a frequent cause of leber congenital amaurosis","volume":"79","author":"Den Hollander","year":"2006","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.mam.2026.101462_bib35","doi-asserted-by":"crossref","first-page":"3983","DOI":"10.1016\/j.cell.2023.07.039","article-title":"Phage-assisted evolution and protein engineering yield compact, efficient prime editors","volume":"186","author":"Doman","year":"2023","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib36","article-title":"In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice","volume":"121","author":"Du","year":"2024"},{"key":"10.1016\/j.mam.2026.101462_bib37","doi-asserted-by":"crossref","first-page":"1818","DOI":"10.1177\/15353702231199069","article-title":"CRISPR\/SaCas9-based gene editing rescues photoreceptor degeneration throughout a rhodopsin-associated autosomal dominant retinitis pigmentosa mouse model","volume":"248","author":"Du","year":"2023","journal-title":"Exp. Biol. Med."},{"key":"10.1016\/j.mam.2026.101462_bib38","doi-asserted-by":"crossref","first-page":"984","DOI":"10.1038\/s41586-024-07552-4","article-title":"Bridge RNAs direct programmable recombination of target and donor DNA","volume":"630","author":"Durrant","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib39","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1007\/s00005-010-0063-4","article-title":"Lentiviral vectors in gene therapy: their current status and future potential","volume":"58","author":"Escors","year":"2010","journal-title":"Arch. Immunol. Ther. Exp."},{"key":"10.1016\/j.mam.2026.101462_bib40","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2023.1138596","article-title":"Immunogenicity of CRISPR therapeutics\u2014Critical considerations for clinical translation","volume":"11","author":"Ewaisha","year":"2023","journal-title":"Front. Bioeng. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib41","doi-asserted-by":"crossref","DOI":"10.1016\/j.exer.2024.110223","article-title":"The vectors went in two-by-two: transduction efficiency and tolerability of dual and triple rAAV vector delivery following intravitreal injection for genome-editing applications","volume":"251","author":"Fehrman","year":"2025","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.mam.2026.101462_bib42","doi-asserted-by":"crossref","first-page":"1842","DOI":"10.1038\/s41467-019-09693-x","article-title":"Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes","volume":"10","author":"Ferdosi","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib43","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pcbi.1007857","article-title":"Towards optogenetic vision restoration with high resolution","volume":"16","author":"Ferrari","year":"2020","journal-title":"PLoS Comput. Biol."},{"key":"10.1016\/j.mam.2026.101462_bib44","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1001\/jamaophthalmol.2020.1032","article-title":"Safety and vision outcomes of subretinal gene therapy targeting cone photoreceptors in achromatopsia: a nonrandomized controlled trial","volume":"138","author":"Fischer","year":"2020","journal-title":"JAMA Ophthalmology"},{"key":"10.1016\/j.mam.2026.101462_bib45","article-title":"P3HT-GRAPHENE device for the restoration of visual properties in a rat model of retinitis pigmentosa","author":"Francia","year":"2022","journal-title":"bioRxiv"},{"key":"10.1016\/j.mam.2026.101462_bib46","doi-asserted-by":"crossref","DOI":"10.1038\/s42003-025-07557-3","article-title":"Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration","volume":"8","author":"Fry","year":"2025","journal-title":"Commun. Biol."},{"key":"10.1016\/j.mam.2026.101462_bib47","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1001\/jamaophthalmol.2020.6418","article-title":"Analysis of pathogenic variants correctable with CRISPR base editing among patients with recessive inherited retinal degeneration","volume":"139","author":"Fry","year":"2021","journal-title":"JAMA Ophthalmology"},{"key":"10.1016\/j.mam.2026.101462_bib48","article-title":"In vivo prime editing rescues photoreceptor degeneration in nonsense mutant retinitis pigmentosa","volume":"16","author":"Fu","year":"2025","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib49","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0079369","article-title":"Unexpected CEP290 mRNA splicing in a humanized Knock-In mouse model for leber congenital amaurosis","volume":"8","author":"Garanto","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.mam.2026.101462_bib50","doi-asserted-by":"crossref","first-page":"15041","DOI":"10.3390\/ijms232315041","article-title":"Optogenetic therapy for visual restoration","volume":"23","author":"Garc\u00eda-Ayuso","year":"2022","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.mam.2026.101462_bib51","doi-asserted-by":"crossref","first-page":"994","DOI":"10.3390\/ijms21030994","article-title":"AAV-mediated gene delivery to 3D retinal organoids derived from human induced pluripotent stem cells","volume":"21","author":"Garita-Hernandez","year":"2020","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.mam.2026.101462_bib52","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u00b7T to G\u00b7C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib53","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1111\/ceo.13917","article-title":"Inherited retinal diseases: therapeutics, clinical trials and end points\u2014A review","volume":"49","author":"Georgiou","year":"2021","journal-title":"Clin. Exp. Ophthalmol."},{"key":"10.1016\/j.mam.2026.101462_bib54","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1093\/hmg\/ddx438","article-title":"Cas9\/sgRNA selective targeting of the P23H rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery","volume":"27","author":"Giannelli","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.mam.2026.101462_bib55","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.cell.2013.06.044","article-title":"CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes","volume":"154","author":"Gilbert","year":"2013","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib56","article-title":"RNA-based therapies in inherited retinal diseases","volume":"14","author":"Girach","year":"2022","journal-title":"Therap. Advan. Ophthalmol."},{"key":"10.1016\/j.mam.2026.101462_bib57","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1038\/s41593-021-00969-4","article-title":"AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset","volume":"25","author":"Goertsen","year":"2022","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.mam.2026.101462_bib58","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1016\/j.exer.2006.08.014","article-title":"Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme","volume":"84","author":"Gorbatyuk","year":"2007","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.mam.2026.101462_bib59","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1038\/s41586-019-1161-z","article-title":"Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors","volume":"569","author":"Gr\u00fcnewald","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib60","doi-asserted-by":"crossref","first-page":"1795","DOI":"10.1016\/S0140-6736(06)69740-7","article-title":"Retinitis pigmentosa","volume":"368","author":"Hartong","year":"2006","journal-title":"Lancet"},{"key":"10.1016\/j.mam.2026.101462_bib61","doi-asserted-by":"crossref","first-page":"994","DOI":"10.1038\/s41586-024-07570-2","article-title":"Structural mechanism of bridge RNA-guided recombination","volume":"630","author":"Hiraizumi","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib62","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1038\/s41551-024-01296-2","article-title":"Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations","volume":"9","author":"Ho\u0142ubowicz","year":"2025","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.mam.2026.101462_bib63","doi-asserted-by":"crossref","first-page":"4008","DOI":"10.1093\/nar\/gkab148","article-title":"Discovery and engineering of small SlugCas9 with broad targeting range and high specificity and activity","volume":"49","author":"Hu","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.mam.2026.101462_bib64","doi-asserted-by":"crossref","first-page":"1375","DOI":"10.2147\/OPTH.S109995","article-title":"Efficacy of valproic acid for retinitis pigmentosa patients: a pilot study","volume":"10","author":"Iraha","year":"2016","journal-title":"Clin. Ophthalmol."},{"key":"10.1016\/j.mam.2026.101462_bib65","doi-asserted-by":"crossref","first-page":"785","DOI":"10.3390\/cells12050785","article-title":"Various AAV serotypes and their applications in gene therapy: an overview","volume":"12","author":"Issa","year":"2023","journal-title":"Cells"},{"key":"10.1016\/j.mam.2026.101462_bib66","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abg2661","article-title":"High-purity production and precise editing of DNA base editing ribonucleoproteins","volume":"7","author":"Jang","year":"2021","journal-title":"Sci. Adv."},{"key":"10.1016\/j.mam.2026.101462_bib67","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib68","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1016\/j.omtn.2022.11.021","article-title":"Visual function restoration in a mouse model of leber congenital amaurosis via therapeutic base editing","volume":"31","author":"Jo","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib69","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aax1210","article-title":"CRISPR-Cas9\u2013mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of leber congenital amaurosis","volume":"5","author":"Jo","year":"2019","journal-title":"Sci. Adv."},{"key":"10.1016\/j.mam.2026.101462_bib70","doi-asserted-by":"crossref","first-page":"674","DOI":"10.1016\/j.omtn.2023.02.020","article-title":"Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C","volume":"31","author":"Kaltak","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib71","doi-asserted-by":"crossref","first-page":"2986","DOI":"10.1038\/s41596-019-0210-2","article-title":"SHERLOCK: nucleic acid detection with CRISPR nucleases","volume":"14","author":"Kellner","year":"2019","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.mam.2026.101462_bib72","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1038\/nmeth.3284","article-title":"Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells","volume":"12","author":"Kim","year":"2015","journal-title":"Nat. Methods"},{"key":"10.1016\/j.mam.2026.101462_bib73","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1126\/science.aat5011","article-title":"CRISPR-cas guides the future of genetic engineering","volume":"361","author":"Knott","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib74","doi-asserted-by":"crossref","first-page":"11300","DOI":"10.1073\/pnas.0604056103","article-title":"Antioxidants reduce cone cell death in a model of retinitis pigmentosa","volume":"103","author":"Komeima","year":"2006","journal-title":"Proc. Natl. Acad. Sci. U. S. A"},{"key":"10.1016\/j.mam.2026.101462_bib75","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib76","doi-asserted-by":"crossref","first-page":"665","DOI":"10.1016\/j.cell.2018.02.033","article-title":"Transcriptome engineering with RNA-targeting type VI-D CRISPR effectors","volume":"173","author":"Konermann","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib77","article-title":"Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina","volume":"121","author":"Kumar","year":"2024"},{"key":"10.1016\/j.mam.2026.101462_bib78","doi-asserted-by":"crossref","first-page":"2001","DOI":"10.1016\/j.ymthe.2021.02.022","article-title":"Engineered prime editors with PAM flexibility","volume":"29","author":"Kweon","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib79","article-title":"In vivo editing of the human mutant rhodopsin gene by electroporation of plasmid-based CRISPR\/Cas9 in the mouse retina. Molecular therapy","volume":"5","author":"Latella","year":"2016","journal-title":"Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib80","doi-asserted-by":"crossref","first-page":"1317","DOI":"10.1038\/s41587-020-0555-7","article-title":"CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity","volume":"38","author":"Lazzarotto","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib81","article-title":"Bystander base editing interferes with visual function restoration in leber congenital amaurosis","author":"Lee","year":"2024","journal-title":"bioRxiv"},{"key":"10.1016\/j.mam.2026.101462_bib82","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.mam.2026.101462_bib83","doi-asserted-by":"crossref","first-page":"817","DOI":"10.1086\/383228","article-title":"Bietti crystalline corneoretinal dystrophy is caused by mutations in the novel gene CYP4V2","volume":"74","author":"Li","year":"2004","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.mam.2026.101462_bib84","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1016\/j.omtm.2018.11.009","article-title":"A self-deleting AAV-CRISPR system for in vivo genome editing","volume":"12","author":"Li","year":"2018","journal-title":"Mol. Ther., Methods Clin. Dev."},{"key":"10.1016\/j.mam.2026.101462_bib85","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1089\/crispr.2017.0009","article-title":"Allele-specific CRISPR-Cas9 genome editing of the single-base P23H mutation for rhodopsin-associated dominant retinitis pigmentosa","volume":"1","author":"Li","year":"2018","journal-title":"CRISPR J."},{"key":"10.1016\/j.mam.2026.101462_bib86","doi-asserted-by":"crossref","first-page":"501","DOI":"10.1016\/j.jcma.2015.05.010","article-title":"Retinal prostheses in degenerative retinal diseases","volume":"78","author":"Lin","year":"2015","journal-title":"J. Chin. Med. Assoc."},{"key":"10.1016\/j.mam.2026.101462_bib87","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.84065","article-title":"Allele-specific gene-editing approach for vision loss restoration in RHO-associated retinitis pigmentosa","volume":"12","author":"Liu","year":"2023","journal-title":"eLife"},{"key":"10.1016\/j.mam.2026.101462_bib88","doi-asserted-by":"crossref","first-page":"3456","DOI":"10.1093\/nar\/gkac153","article-title":"Global chromosome rearrangement induced by CRISPR-Cas9 reshapes the genome and transcriptome of human cells","volume":"50","author":"Liu","year":"2022","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.mam.2026.101462_bib89","article-title":"Efficient rescue of retinal degeneration in Pde6a mice by engineered base editing and prime editing","author":"Liu","year":"2024","journal-title":"Adv. Sci."},{"key":"10.1016\/j.mam.2026.101462_bib90","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2023.1339189","article-title":"Beyond the promise: evaluating and mitigating off-target effects in CRISPR gene editing for safer therapeutics","volume":"11","author":"Lopes","year":"2024","journal-title":"Front. Bioeng. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib91","doi-asserted-by":"crossref","first-page":"1129","DOI":"10.1016\/S0140-6736(13)62117-0","article-title":"Retinal gene therapy in patients with choroideremia: initial findings from a phase 1\/2 clinical trial","volume":"383","author":"MacLaren","year":"2014","journal-title":"Lancet"},{"key":"10.1016\/j.mam.2026.101462_bib92","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/s41591-018-0327-9","article-title":"Development of a gene-editing approach to restore vision loss in leber congenital amaurosis type 10","volume":"25","author":"Maeder","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.mam.2026.101462_bib93","doi-asserted-by":"crossref","first-page":"1460","DOI":"10.1016\/j.ophtha.2021.03.031","article-title":"Durability of voretigene neparvovec for biallelic RPE65-Mediated inherited retinal disease: phase 3 results at 3 and 4 years","volume":"128","author":"Maguire","year":"2021","journal-title":"Ophthalmology"},{"key":"10.1016\/j.mam.2026.101462_bib94","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for leber's congenital amaurosis","volume":"358","author":"Maguire","year":"2008","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.mam.2026.101462_bib95","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1126\/science.1232033","article-title":"RNA-guided human genome engineering via Cas9","volume":"339","author":"Mali","year":"2013","journal-title":"Science"},{"issue":"6 16","key":"10.1016\/j.mam.2026.101462_bib97","doi-asserted-by":"crossref","first-page":"681","DOI":"10.1038\/nmat4874","article-title":"A fully organic retinal prosthesis restores vision in a rat model of degenerative blindness","volume":"16","author":"Maya-Vetencourt","year":"2017","journal-title":"Nat. Mater."},{"key":"10.1016\/j.mam.2026.101462_bib98","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2020.570909","article-title":"Optogenetic gene therapy for the degenerate retina: recent advances","volume":"14","author":"McClements","year":"2020","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.mam.2026.101462_bib99","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1167\/tvst.11.4.3","article-title":"Tropism of AAV vectors in photoreceptor-like cells of human iPSC-Derived retinal organoids","volume":"11","author":"McClements","year":"2022","journal-title":"Transl. Vis. Sci. Technol."},{"issue":"1 15","key":"10.1016\/j.mam.2026.101462_bib100","first-page":"1","article-title":"In vivo genome editing via CRISPR\/Cas9-mediated homology-independent targeted integration for bietti crystalline corneoretinal dystrophy treatment","volume":"15","author":"Meng","year":"2024","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib101","doi-asserted-by":"crossref","first-page":"642","DOI":"10.1038\/mt.2010.293","article-title":"Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa","volume":"19","author":"Millington-Ward","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib102","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1089\/crispr.2023.0065","article-title":"Rapid variant pathogenicity analysis by CRISPR activation of CRB1 gene expression in patient-derived fibroblasts","volume":"7","author":"Moon","year":"2024","journal-title":"CRISPR J."},{"key":"10.1016\/j.mam.2026.101462_bib103","doi-asserted-by":"crossref","first-page":"1818","DOI":"10.1016\/j.ymthe.2018.04.017","article-title":"In situ gene therapy via AAV-CRISPR-Cas9-Mediated targeted gene regulation","volume":"26","author":"Moreno","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib104","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/s41591-024-03422-8","article-title":"High-efficiency base editing in the retina in primates and human tissues","volume":"31","author":"Muller","year":"2025","journal-title":"Nat. Med."},{"key":"10.1016\/j.mam.2026.101462_bib105","doi-asserted-by":"crossref","first-page":"118","DOI":"10.1002\/emmm.201000119","article-title":"Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa","volume":"3","author":"Mussolino","year":"2011","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.mam.2026.101462_bib106","doi-asserted-by":"crossref","DOI":"10.3390\/cells9071608","article-title":"Latest developed strategies to minimize the off-target effects in CRISPR-Cas-Mediated genome editing","volume":"9","author":"Naeem","year":"2020","journal-title":"Cells"},{"issue":"1 5","key":"10.1016\/j.mam.2026.101462_bib107","first-page":"1","article-title":"Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR\/Cas9","volume":"5","author":"Nakade","year":"2014","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib108","doi-asserted-by":"crossref","first-page":"2653","DOI":"10.1182\/blood-2005-10-4035","article-title":"Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver","volume":"107","author":"Nathwani","year":"2006","journal-title":"Blood"},{"key":"10.1016\/j.mam.2026.101462_bib109","doi-asserted-by":"crossref","first-page":"eaaz6893","DOI":"10.1126\/sciadv.aaz6893","article-title":"Impact of mRNA chemistry and manufacturing process on innate immune activation","volume":"6","author":"Nelson","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.mam.2026.101462_bib110","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1038\/s41587-021-01039-7","article-title":"Engineered pegRNAs improve prime editing efficiency","volume":"40","author":"Nelson","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib111","doi-asserted-by":"crossref","DOI":"10.1167\/tvst.11.5.24","article-title":"Restoration of vision and retinal responses after adeno-associated virus\u2013mediated optogenetic therapy in blind dogs","volume":"11","author":"Nikonov","year":"2022","journal-title":"Transl. Vis. Sci. Technol."},{"key":"10.1016\/j.mam.2026.101462_bib112","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-019-14181-3","article-title":"Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice","volume":"11","author":"Nishiguchi","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib113","article-title":"CRISPR editing of anti-anemia drug target rescues independent preclinical models of retinitis pigmentosa","volume":"5","author":"Nolan","year":"2024","journal-title":"Cell Rep. Med."},{"key":"10.1016\/j.mam.2026.101462_bib114","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1038\/s42003-021-01765-3","article-title":"Photoreceptor metabolic reprogramming: current understanding and therapeutic implications","volume":"4","author":"Pan","year":"2021","journal-title":"Commun. Biol."},{"key":"10.1016\/j.mam.2026.101462_bib115","doi-asserted-by":"crossref","DOI":"10.3389\/fncel.2023.1236826","article-title":"Current approaches to vision restoration using optogenetic therapy","volume":"17","author":"Parnami","year":"2023","journal-title":"Front. Cell. Neurosci."},{"key":"10.1016\/j.mam.2026.101462_bib116","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1016\/j.ajhg.2021.01.006","article-title":"Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model","volume":"108","author":"Patrizi","year":"2021","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.mam.2026.101462_bib117","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-024-47800-9","article-title":"CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing","volume":"15","author":"Pedrazzoli","year":"2024","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib118","article-title":"Exon 13-skipped USH2A protein retains functional integrity in mice, suggesting an exo-skipping therapeutic approach to treat USH2A-associated disease","author":"Pendse","year":"2020","journal-title":"bioRxiv"},{"key":"10.1016\/j.mam.2026.101462_bib119","doi-asserted-by":"crossref","DOI":"10.1126\/science.adz0276","article-title":"Megabase-scale human genome rearrangement with programmable bridge recombinases","author":"Perry","year":"2025","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib120","doi-asserted-by":"crossref","DOI":"10.21037\/atm.2016.11.24","article-title":"Are long stimulus pulse durations the answer to improving spatial resolution in retinal prostheses?","volume":"4","author":"Petoe","year":"2016","journal-title":"Ann. Transl. Med."},{"key":"10.1016\/j.mam.2026.101462_bib121","first-page":"4501","article-title":"In-frame exon skipping fails to rescue retinal or hair cell defects in zebrafish models of PCDH15 (USH1F)","volume":"58","author":"Phillips","year":"2017","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.mam.2026.101462_bib122","doi-asserted-by":"crossref","first-page":"1972","DOI":"10.1056\/NEJMoa2309915","article-title":"Gene editing for CEP290 -Associated retinal degeneration","volume":"390","author":"Pierce","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.mam.2026.101462_bib123","doi-asserted-by":"crossref","first-page":"429","DOI":"10.3390\/biomedicines13020429","article-title":"Evolution of light-sensitive proteins in optogenetic approaches for vision restoration: a comprehensive review","volume":"13","author":"Pobo\u017cy","year":"2025","journal-title":"Biomedicines"},{"key":"10.1016\/j.mam.2026.101462_bib124","doi-asserted-by":"crossref","first-page":"269","DOI":"10.3390\/biom12020269","article-title":"Advances in ophthalmic optogenetics: approaches and applications","volume":"12","author":"Prosseda","year":"2022","journal-title":"Biomolecules"},{"key":"10.1016\/j.mam.2026.101462_bib125","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1038\/nn.2234","article-title":"Stimulation of the insulin\/mTOR pathway delays cone death in a mouse model of retinitis pigmentosa","volume":"12","author":"Punzo","year":"2009","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.mam.2026.101462_bib126","doi-asserted-by":"crossref","first-page":"1173","DOI":"10.1016\/j.cell.2013.02.022","article-title":"Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression","volume":"152","author":"Qi","year":"2013","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib127","doi-asserted-by":"crossref","DOI":"10.1084\/jem.20220776","article-title":"Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas","volume":"220","author":"Qin","year":"2023","journal-title":"J. Exp. Med."},{"key":"10.1016\/j.mam.2026.101462_bib128","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1038\/nature14299","article-title":"In vivo genome editing using Staphylococcus aureus Cas9","volume":"520","author":"Ran","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib129","doi-asserted-by":"crossref","first-page":"770","DOI":"10.1038\/s41576-018-0059-1","article-title":"Base editing: precision chemistry on the genome and transcriptome of living cells","volume":"19","author":"Rees","year":"2018","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.mam.2026.101462_bib131","doi-asserted-by":"crossref","first-page":"883","DOI":"10.1038\/s41587-020-0453-z","article-title":"Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity","volume":"38","author":"Richter","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.mam.2026.101462_bib132","doi-asserted-by":"crossref","first-page":"781","DOI":"10.1038\/s41596-021-00666-3","article-title":"dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy","volume":"17","author":"Riedmayr","year":"2022","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.mam.2026.101462_bib133","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1007\/978-1-0716-2772-3_2","article-title":"Viltolarsen: from preclinical studies to FDA approval","volume":"2587","author":"Roshmi","year":"2023","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.mam.2026.101462_bib134","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ymthe.2016.12.006","article-title":"CRISPR\/Cas9-Mediated genome editing as a therapeutic approach for leber congenital amaurosis 10","volume":"25","author":"Ruan","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib135","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet (London, England)"},{"key":"10.1016\/j.mam.2026.101462_bib136","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a017111","article-title":"Clinical characteristics and current therapies for inherited retinal degenerations","volume":"5","author":"Sahel","year":"2015","journal-title":"Cold Spring Harb. Perspect. Med."},{"key":"10.1016\/j.mam.2026.101462_bib137","doi-asserted-by":"crossref","first-page":"3683","DOI":"10.1093\/nar\/gkh703","article-title":"Conservative homologous recombination preferentially repairs DNA double-strand breaks in the S phase of the cell cycle in human cells","volume":"32","author":"Saleh-Gohari","year":"2004","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.mam.2026.101462_bib138","doi-asserted-by":"crossref","DOI":"10.1016\/j.exer.2022.108961","article-title":"RPE based gene and cell therapy for inherited retinal diseases: a review","volume":"217","author":"Sanie-Jahromi","year":"2022","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.mam.2026.101462_bib139","doi-asserted-by":"crossref","first-page":"9275","DOI":"10.1093\/nar\/gkr606","article-title":"The Streptococcus thermophilus CRISPR\/Cas system provides immunity in Escherichia coli","volume":"39","author":"Sapranauskas","year":"2011","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.mam.2026.101462_bib140","doi-asserted-by":"crossref","DOI":"10.3390\/ijms22179154","article-title":"Zebrafish as a model to evaluate a CRISPR\/Cas9-Based exon excision approach as a future treatment option for EYS-associated retinitis pigmentosa","author":"Schellens","year":"2021","journal-title":"Int. J. Mol. Sci. Articl."},{"key":"10.1016\/j.mam.2026.101462_bib141","doi-asserted-by":"crossref","first-page":"980","DOI":"10.1016\/j.omtn.2023.05.020","article-title":"A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa. Molecular therapy","volume":"32","author":"Schellens","year":"2023","journal-title":"Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib142","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1038\/s41589-023-01427-x","article-title":"Continuous directed evolution of a compact CjCas9 variant with broad PAM compatibility","volume":"20","author":"Schmidheini","year":"2024","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.mam.2026.101462_bib143","doi-asserted-by":"crossref","DOI":"10.1038\/s41392-022-01234-1","article-title":"Dual-AAV split prime editor corrects the mutation and phenotype in mice with inherited retinal degeneration","volume":"8","author":"She","year":"2023","journal-title":"Signal Transduct. Targeted Ther."},{"key":"10.1016\/j.mam.2026.101462_bib144","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1007\/s40265-021-01512-2","article-title":"Casimersen: first approval","volume":"81","author":"Shirley","year":"2021","journal-title":"Drugs"},{"key":"10.1016\/j.mam.2026.101462_bib145","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1038\/s41586-025-09021-y","article-title":"Custom CRISPR\u2013Cas9 PAM variants via scalable engineering and machine learning","volume":"643","author":"Silverstein","year":"2025","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib146","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1016\/j.omtm.2018.06.006","article-title":"Prevalence of pre-existing antibodies to CRISPR-associated nuclease Cas9 in the USA population","volume":"10","author":"Simhadri","year":"2018","journal-title":"Mol. Ther., Methods Clin. Dev."},{"key":"10.1016\/j.mam.2026.101462_bib147","doi-asserted-by":"crossref","DOI":"10.1038\/mtna.2016.89","article-title":"Antisense oligonucleotide-based splice correction for USH2A-associated retinal degeneration caused by a frequent deep-intronic mutation","volume":"5","author":"Slijkerman","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib148","doi-asserted-by":"crossref","first-page":"2422","DOI":"10.1016\/j.ymthe.2023.06.019","article-title":"Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs","volume":"31","author":"Stahl","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib149","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2023.02.011","article-title":"In vivo base editing rescues photoreceptors in a mouse model of retinitis pigmentosa","author":"Su","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib150","doi-asserted-by":"crossref","DOI":"10.1038\/s41551-020-00632-6","article-title":"Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing","volume":"5","author":"Suh","year":"2020","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.mam.2026.101462_bib151","author":"Sun"},{"key":"10.1016\/j.mam.2026.101462_bib152","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1038\/nature20565","article-title":"In vivo genome editing via CRISPR\/Cas9 mediated homology-independent targeted integration","volume":"540","author":"Suzuki","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib153","doi-asserted-by":"crossref","first-page":"563","DOI":"10.1038\/nrn2880","article-title":"Transcriptional regulation of photoreceptor development and homeostasis in the Mammalian retina","volume":"11","author":"Swaroop","year":"2010","journal-title":"Nat. Rev. Neurosci."},{"key":"10.1016\/j.mam.2026.101462_bib154","doi-asserted-by":"crossref","first-page":"1466","DOI":"10.1016\/j.ymthe.2025.01.042","article-title":"Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in USH2A and comparison of methods","volume":"33","author":"Tachida","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib155","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1016\/j.cell.2014.09.039","article-title":"A protein-tagging system for signal amplification in gene expression and fluorescence imaging","volume":"159","author":"Tanenbaum","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib156","doi-asserted-by":"crossref","first-page":"6423","DOI":"10.1093\/nar\/gkac506","article-title":"Bi-PE: bi-directional priming improves CRISPR\/Cas9 prime editing in mammalian cells","volume":"50","author":"Tao","year":"2022","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.mam.2026.101462_bib157","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1016\/j.medj.2023.05.005","article-title":"Massively parallel CRISPR off-target detection enables rapid off-target prediction model building","volume":"4","author":"Tian","year":"2023","journal-title":"Med"},{"key":"10.1016\/j.mam.2026.101462_bib158","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1038\/s44321-023-00008-8","article-title":"Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1","volume":"16","author":"Torella","year":"2024","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.mam.2026.101462_bib159","doi-asserted-by":"crossref","first-page":"1421","DOI":"10.1016\/j.ophtha.2018.04.001","article-title":"CRISPR-based genome surgery for the treatment of autosomal dominant retinitis pigmentosa","volume":"125","author":"Tsai","year":"2018","journal-title":"Ophthalmology"},{"key":"10.1016\/j.mam.2026.101462_bib161","doi-asserted-by":"crossref","first-page":"1446","DOI":"10.1172\/JCI79766","article-title":"Activated mTORC1 promotes long-term cone survival in retinitis pigmentosa mice","volume":"125","author":"Venkatesh","year":"2015","journal-title":"J. Clin. Investig."},{"key":"10.1016\/j.mam.2026.101462_bib162","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/j.preteyeres.2018.03.005","article-title":"Non-syndromic retinitis pigmentosa","volume":"66","author":"Verbakel","year":"2018","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.mam.2026.101462_bib163","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1089\/hum.2020.321","article-title":"Optimized adeno-associated virus vectors for efficient transduction of human retinal organoids","volume":"32","author":"V\u00f6lkner","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.mam.2026.101462_bib164","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1126\/science.aba8853","article-title":"Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants","volume":"368","author":"Walton","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.mam.2026.101462_bib165","author":"Weber"},{"key":"10.1016\/j.mam.2026.101462_bib166","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aac4877","article-title":"Improving the spatial resolution of epiretinal implants by increasing stimulus pulse duration","volume":"7","author":"Weitz","year":"2015","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.mam.2026.101462_bib167","doi-asserted-by":"crossref","first-page":"5782","DOI":"10.3390\/s23135782","article-title":"Retinal prostheses: engineering and clinical perspectives for vision restoration","volume":"23","author":"Wu","year":"2023","journal-title":"Sensors"},{"key":"10.1016\/j.mam.2026.101462_bib168","doi-asserted-by":"crossref","first-page":"1407","DOI":"10.1016\/j.ymthe.2022.02.010","article-title":"CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa","volume":"30","author":"Wu","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib169","doi-asserted-by":"crossref","DOI":"10.1038\/mt.2016.107","article-title":"CRISPR repair reveals causative mutation in a preclinical model of retinitis pigmentosa","volume":"24","author":"Wu","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib170","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1016\/j.bbrc.2011.04.112","article-title":"Identification of CYP4V2 mutation in 21 families and overview of mutation spectrum in bietti crystalline corneoretinal dystrophy","volume":"409","author":"Xiao","year":"2011","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib171","doi-asserted-by":"crossref","first-page":"380","DOI":"10.1038\/nature13589","article-title":"CRISPR-mediated direct mutation of cancer genes in the mouse liver","volume":"514","author":"Xue","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib172","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1038\/s41586-024-07259-6","article-title":"Improving prime editing with an endogenous small RNA-binding protein","volume":"628","author":"Yan","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.mam.2026.101462_bib173","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1016\/j.omtn.2023.08.002","article-title":"Treatment of autosomal dominant retinitis pigmentosa caused by RHO-P23H mutation with high-fidelity Cas13X in mice","volume":"33","author":"Yan","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.mam.2026.101462_bib174","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1038\/s41592-018-0048-5","article-title":"An enhanced CRISPR repressor for targeted mammalian gene regulation","volume":"15","author":"Yeo","year":"2018","journal-title":"Nat. Methods"},{"key":"10.1016\/j.mam.2026.101462_bib175","doi-asserted-by":"crossref","first-page":"2256","DOI":"10.1016\/j.cell.2023.03.034","article-title":"Prediction of efficiencies for diverse prime editing systems in multiple cell types","volume":"186","author":"Yu","year":"2023","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib176","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/ncomms14716","article-title":"Nrl knockdown by AAV-delivered CRISPR\/Cas9 prevents retinal degeneration in mice","volume":"8","author":"Yu","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.mam.2026.101462_bib177","doi-asserted-by":"crossref","first-page":"212","DOI":"10.1016\/j.cell.2018.09.001","article-title":"Structural basis for the RNA-guided ribonuclease activity of CRISPR-Cas13d","volume":"175","author":"Zhang","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib178","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.ymthe.2021.07.011","article-title":"Dual-AAV delivering split prime editor system for in vivo genome editing","volume":"30","author":"Zhi","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.mam.2026.101462_bib179","doi-asserted-by":"crossref","first-page":"835","DOI":"10.1093\/nsr\/nwaa033","article-title":"CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration","volume":"7","author":"Zhou","year":"2020","journal-title":"Natl. Sci. Rev."},{"key":"10.1016\/j.mam.2026.101462_bib180","doi-asserted-by":"crossref","first-page":"590","DOI":"10.1016\/j.cell.2020.03.024","article-title":"Glia-to-Neuron conversion by CRISPR-CasRx alleviates symptoms of neurological disease in mice","volume":"181","author":"Zhou","year":"2020","journal-title":"Cell"},{"key":"10.1016\/j.mam.2026.101462_bib181","doi-asserted-by":"crossref","first-page":"830","DOI":"10.1038\/cr.2017.57","article-title":"Gene and mutation independent therapy via CRISPR-Cas9 mediated cellular reprogramming in rod photoreceptors","volume":"27","author":"Zhu","year":"2017","journal-title":"Cell Res."}],"container-title":["Molecular Aspects of Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S009829972600018X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S009829972600018X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T13:45:45Z","timestamp":1773323145000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S009829972600018X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4]]},"references-count":178,"alternative-id":["S009829972600018X"],"URL":"https:\/\/doi.org\/10.1016\/j.mam.2026.101462","relation":{},"ISSN":["0098-2997"],"issn-type":[{"value":"0098-2997","type":"print"}],"subject":[],"published":{"date-parts":[[2026,4]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"CRISPR as a therapeutic tool for inherited retinal degenerations: Advances, challenges, and future directions","name":"articletitle","label":"Article Title"},{"value":"Molecular Aspects of Medicine","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.mam.2026.101462","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 Elsevier Ltd. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}],"article-number":"101462"}}