{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T03:46:05Z","timestamp":1792813565993,"version":"4.5.0"},"reference-count":48,"publisher":"Association for Research in Vision and Ophthalmology (ARVO)","issue":"13","license":[{"start":{"date-parts":[[2024,11,18]],"date-time":"2024-11-18T00:00:00Z","timestamp":1731888000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Invest. Ophthalmol. Vis. Sci."],"published-print":{"date-parts":[[2024,11,18]]},"DOI":"10.1167\/iovs.65.13.38","type":"journal-article","created":{"date-parts":[[2024,11,18]],"date-time":"2024-11-18T10:31:03Z","timestamp":1731925863000},"page":"38","source":"Crossref","is-referenced-by-count":5,"title":["Optimization of HITI-Mediated Gene Insertion for Rhodopsin and Peripherin-2 in Mouse Rod Photoreceptors: Targeting Dominant Retinitis Pigmentosa"],"prefix":"10.1167","volume":"65","author":[{"given":"Akishi","family":"Onishi","sequence":"first","affiliation":[{"name":"Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Cell and Gene Therapy in Ophthalmology Laboratory, RIKEN Baton Zone Program, Kobe, Japan"},{"name":"Kobe City Eye Hospital Research Center, Kobe, Japan"},{"name":"VCGT Inc., Kobe, Japan"},{"name":"Research Organization of Science and Technology, Ritsumeikan University, Shiga, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuji","family":"Tsunekawa","sequence":"additional","affiliation":[{"name":"Laboratory for Cell Asymmetry, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michiko","family":"Mandai","sequence":"additional","affiliation":[{"name":"Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Kobe City Eye Hospital Research Center, Kobe, Japan"},{"name":"Research Organization of Science and Technology, Ritsumeikan University, Shiga, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aiko","family":"Ishimaru","sequence":"additional","affiliation":[{"name":"Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"VCGT Inc., Kobe, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoko","family":"Ohigashi","sequence":"additional","affiliation":[{"name":"Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Cell and Gene Therapy in Ophthalmology Laboratory, RIKEN Baton Zone Program, Kobe, Japan"},{"name":"Vision Care Inc., Kobe, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Junki","family":"Sho","sequence":"additional","affiliation":[{"name":"Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Cell and Gene Therapy in Ophthalmology Laboratory, RIKEN Baton Zone Program, Kobe, Japan"},{"name":"Vision Care Inc., Kobe, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazushi","family":"Yasuda","sequence":"additional","affiliation":[{"name":"Cell and Gene Therapy in Ophthalmology Laboratory, RIKEN Baton Zone Program, Kobe, Japan"},{"name":"VCGT Inc., Kobe, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keiichiro","family":"Suzuki","sequence":"additional","affiliation":[{"name":"Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, California, United States"},{"name":"Institute for Advanced Co-Creation Studies, Osaka University, Suita, Japan"},{"name":"Graduate School of Engineering Science, Osaka University, Toyonaka, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan Carlos","family":"Izpisua Belmonte","sequence":"additional","affiliation":[{"name":"Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, California, United States"},{"name":"Altos Labs, Inc., San Diego, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fumio","family":"Matsuzaki","sequence":"additional","affiliation":[{"name":"Laboratory for Cell Asymmetry, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Department of Aging Science and Medicine, Medical Innovation Center, Graduate School of Medicine, Kyoto University, Kyoto, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masayo","family":"Takahashi","sequence":"additional","affiliation":[{"name":"Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan"},{"name":"Cell and Gene Therapy in Ophthalmology Laboratory, RIKEN Baton Zone Program, Kobe, Japan"},{"name":"Kobe City Eye Hospital Research Center, Kobe, Japan"},{"name":"VCGT Inc., Kobe, Japan"},{"name":"Vision Care Inc., Kobe, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"27","reference":[{"key":"bib2","doi-asserted-by":"publisher","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"},{"issue":"10","key":"bib3","doi-asserted-by":"publisher","first-page":"1384","DOI":"10.1016\/j.ophtha.2020.04.008","article-title":"Genetic basis of inherited retinal disease in a molecularly characterized cohort of more than 3000 families from the United Kingdom","volume":"127","author":"Pontikos","year":"2020","journal-title":"Ophthalmology"},{"issue":"10","key":"bib4","doi-asserted-by":"publisher","first-page":"662","DOI":"10.1136\/jmedgenet-2018-105691","article-title":"Genetic characteristics of retinitis pigmentosa in 1204 Japanese patients","volume":"56","author":"Koyanagi","year":"2019","journal-title":"J Med Genet"},{"issue":"9","key":"bib5","doi-asserted-by":"publisher","first-page":"1780","DOI":"10.1016\/S0161-6420(99)90342-1","article-title":"Visual acuity impairment in patients with retinitis pigmentosa at age 45 years or older","volume":"106","author":"Grover","year":"1999","journal-title":"Ophthalmology"},{"issue":"1","key":"bib6","doi-asserted-by":"publisher","first-page":"40","DOI":"10.1186\/1750-1172-1-40","article-title":"Retinitis pigmentosa","volume":"1","author":"Hamel","year":"2006","journal-title":"Orphanet J Rare Dis"},{"key":"bib7","doi-asserted-by":"publisher","first-page":"101029","DOI":"10.1016\/j.preteyeres.2021.101029","article-title":"Inherited retinal diseases: linking genes, disease-causing variants, and relevant therapeutic modalities","volume":"89","author":"Schneider","year":"2022","journal-title":"Prog Retin Eye Res"},{"key":"bib8","doi-asserted-by":"publisher","first-page":"113324","DOI":"10.1016\/j.biopha.2022.113324","article-title":"Gene therapy clinical trials, where do we go? An overview","volume":"153","author":"Arabi","year":"2022","journal-title":"Biomed Pharmacother"},{"issue":"9","key":"bib9","doi-asserted-by":"publisher","first-page":"4534","DOI":"10.3390\/ijms22094534","article-title":"An update on gene therapy for inherited retinal dystrophy: experience in Leber congenital amaurosis clinical trials","volume":"22","author":"Chiu","year":"2021","journal-title":"Int J Mol Sci."},{"issue":"4","key":"bib10","doi-asserted-by":"publisher","first-page":"444","DOI":"10.1080\/08164622.2021.1880863","article-title":"Gene therapy for inherited retinal diseases: progress and possibilities","volume":"104","author":"Hu","year":"2021","journal-title":"Clin Exp Optom"},{"key":"bib11","doi-asserted-by":"publisher","first-page":"2909","DOI":"10.2147\/OPTH.S370032","article-title":"Current and future treatment of retinitis pigmentosa","volume":"16","author":"Cross","year":"2022","journal-title":"Clin Ophthalmol"},{"issue":"5","key":"bib12","doi-asserted-by":"publisher","first-page":"2318","DOI":"10.3390\/ijms22052318","article-title":"The landscape of non-viral gene augmentation strategies for inherited retinal diseases","volume":"22","author":"Toualbi","year":"2021","journal-title":"Int J Mol Sci."},{"issue":"9","key":"bib13","doi-asserted-by":"publisher","first-page":"1223","DOI":"10.1136\/bjo-2022-321903","article-title":"Genetic treatment for autosomal dominant inherited retinal dystrophies: approaches, challenges and targeted genotypes","volume":"107","author":"Daich Varela","year":"2023","journal-title":"Br J Ophthalmol"},{"issue":"3","key":"bib14","doi-asserted-by":"publisher","first-page":"396","DOI":"10.1016\/j.tibtech.2022.12.022","article-title":"The genome editing revolution","volume":"41","author":"van der Oost","year":"2023","journal-title":"Trends Biotechnol"},{"issue":"6629","key":"bib15","first-page":"eadd8643","article-title":"CRISPR technology: a decade of genome editing is only the beginning","volume":"379","author":"Wang","year":"2023","journal-title":"Science (1979)"},{"issue":"6","key":"bib16","doi-asserted-by":"publisher","first-page":"849","DOI":"10.1007\/s12033-022-00639-1","article-title":"A review on advanced CRISPR-based genome-editing tools: base editing and prime editing","volume":"65","author":"Saber Sichani","year":"2023","journal-title":"Mol Biotechnol"},{"key":"bib17","doi-asserted-by":"publisher","first-page":"737632","DOI":"10.3389\/fgeed.2021.737632","article-title":"Progress in gene editing tools and their potential for correcting mutations underlying hearing and vision loss","volume":"3","author":"Botto","year":"2021","journal-title":"Front Genome Ed"},{"issue":"10","key":"bib18","doi-asserted-by":"publisher","first-page":"2542","DOI":"10.3390\/ijms20102542","article-title":"Genome editing as a treatment for the most prevalent causative genes of autosomal dominant retinitis pigmentosa","volume":"20","author":"Diakatou","year":"2019","journal-title":"Int J Mol Sci"},{"issue":"2","key":"bib19","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1038\/s10038-017-0352-4","article-title":"In vivo genome editing via the HITI method as a tool for gene therapy","volume":"63","author":"Suzuki","year":"2017","journal-title":"J Hum Genet"},{"issue":"7631","key":"bib20","doi-asserted-by":"publisher","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"},{"issue":"1","key":"bib21","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1038\/s41467-022-29550-8","article-title":"Therapeutic homology-independent targeted integration in retina and liver","volume":"13","author":"Tornabene","year":"2022","journal-title":"Nat Commun."},{"issue":"1","key":"bib22","first-page":"1","article-title":"Mutation-independent gene knock-in therapy targeting 5'UTR for autosomal dominant retinitis pigmentosa","volume":"8","author":"Hoang","year":"2023","journal-title":"Signal Transduct Target Ther."},{"key":"bib23","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.preteyeres.2017.10.002","article-title":"The molecular and cellular basis of rhodopsin retinitis pigmentosa reveals potential strategies for therapy","volume":"62","author":"Athanasiou","year":"2018","journal-title":"Prog Retin Eye Res"},{"key":"bib24","doi-asserted-by":"publisher","first-page":"1132179","DOI":"10.3389\/fnins.2023.1132179","article-title":"Rhodopsin-associated retinal dystrophy: disease mechanisms and therapeutic strategies","volume":"17","author":"Zhen","year":"2023","journal-title":"Front Neurosci"},{"issue":"2","key":"bib26","doi-asserted-by":"publisher","first-page":"234","DOI":"10.1016\/j.neuron.2008.12.006","article-title":"Pias3-dependent SUMOylation directs rod photoreceptor development","volume":"61","author":"Onishi","year":"2009","journal-title":"Neuron"},{"issue":"20","key":"bib27","doi-asserted-by":"publisher","first-page":"11372","DOI":"10.1128\/JVI.01327-07","article-title":"Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors","volume":"81","author":"Allocca","year":"2007","journal-title":"J Virol"},{"issue":"5","key":"bib28","doi-asserted-by":"publisher","first-page":"662","DOI":"10.1016\/j.stemcr.2014.03.011","article-title":"Transplantation of embryonic and induced pluripotent stem cell-derived 3D retinal sheets into retinal degenerative mice","volume":"2","author":"Assawachananont","year":"2014","journal-title":"Stem Cell Reports"},{"issue":"9","key":"bib29","doi-asserted-by":"publisher","first-page":"1059","DOI":"10.1038\/nn.2618","article-title":"Pias3-dependent SUMOylation controls mammalian cone photoreceptor differentiation","volume":"13","author":"Onishi","year":"2010","journal-title":"Nat Neurosci"},{"issue":"2","key":"bib30","doi-asserted-by":"publisher","first-page":"776","DOI":"10.1167\/iovs.17-22406","article-title":"Culture systems of dissociated mouse and human pluripotent stem cell-derived retinal ganglion cells purified by two-step immunopanning","volume":"59","author":"Kobayashi","year":"2018","journal-title":"Invest Ophthalmol Vis Sci"},{"issue":"1","key":"bib31","doi-asserted-by":"publisher","first-page":"300","DOI":"10.1152\/jn.00055.2017","article-title":"Comparison of optomotor and optokinetic reflexes in mice","volume":"118","author":"Kretschmer","year":"2017","journal-title":"J Neurophysiol"},{"issue":"C","key":"bib33","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/B978-0-12-185279-5.50013-3","article-title":"Preparing rod outer segment membranes, regenerating rhodopsin, and determining rhodopsin concentration","volume":"15","author":"McDowell","year":"1993","journal-title":"Meth Neurosci"},{"issue":"3","key":"bib34","doi-asserted-by":"publisher","first-page":"836","DOI":"10.1073\/pnas.85.3.836","article-title":"Introns increase transcriptional efficiency in transgenic mice","volume":"85","author":"Brinster","year":"1988","journal-title":"Proc Natl Acad Sci USA"},{"issue":"10","key":"bib35","first-page":"4395","article-title":"Comparison of intron-dependent and intron-independent gene expression","volume":"8","author":"Buchman","year":"1988","journal-title":"Mol Cell Biol"},{"issue":"6","key":"bib36","first-page":"3070","article-title":"A generic intron increases gene expression in transgenic mice","volume":"11","author":"Choi","year":"1991","journal-title":"Mol Cell Biol"},{"issue":"1","key":"bib37","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1085\/jgp.200609729","article-title":"Physiological properties of rod photoreceptor cells in green-sensitive cone pigment knock-in mice","volume":"130","author":"Sakurai","year":"2007","journal-title":"J Gen Physiol"},{"issue":"6","key":"bib38","doi-asserted-by":"publisher","first-page":"859","DOI":"10.1016\/j.exer.2004.12.017","article-title":"Expression and localization of an exogenous G protein-coupled receptor fused with the rhodopsin C-terminal sequence in the retinal rod cells of knockin mice","volume":"80","author":"Kodama","year":"2005","journal-title":"Exp Eye Res"},{"issue":"33","key":"bib39","doi-asserted-by":"publisher","first-page":"20563","DOI":"10.1016\/S0021-9258(17)30539-2","article-title":"Mouse opsin. Gene structure and molecular basis of multiple transcripts","volume":"265","author":"Al-Ubaidi","year":"1990","journal-title":"J Biol Chem"},{"issue":"17","key":"bib40","doi-asserted-by":"publisher","first-page":"3216","DOI":"10.1242\/dev.136325","article-title":"Developing a de novo targeted knock-in method based on in utero electroporation into the mammalian brain","volume":"143","author":"Tsunekawa","year":"2016","journal-title":"Development (Cambridge)"},{"key":"bib41","doi-asserted-by":"publisher","first-page":"309","DOI":"10.1007\/978-1-0716-1441-9","article-title":"Protocol for de novo gene targeting via in utero electroporation","volume":"2312","author":"Tsunekawa","year":"2021","journal-title":"Meth Mol Biol"},{"issue":"3","key":"bib42","doi-asserted-by":"publisher","first-page":"1027","DOI":"10.1073\/pnas.0610155104","article-title":"Controlled expression of transgenes introduced by in vivo electroporation","volume":"104","author":"Matsuda","year":"2007","journal-title":"Proc Natl Acad Sci USA"},{"issue":"12","key":"bib43","doi-asserted-by":"publisher","first-page":"10551","DOI":"10.1074\/jbc.M110.209759","article-title":"Probing mechanisms of photoreceptor degeneration in a new mouse model of the common form of autosomal dominant retinitis pigmentosa due to P23H opsin mutations","volume":"286","author":"Sakami","year":"2011","journal-title":"J Biol Chem"},{"issue":"7","key":"bib44","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1093\/hmg\/ddt561","article-title":"P23H opsin knock-in mice reveal a novel step in retinal rod disc morphogenesis","volume":"23","author":"Sakami","year":"2014","journal-title":"Hum Mol Genet"},{"issue":"18","key":"bib45","doi-asserted-by":"publisher","first-page":"8732","DOI":"10.1128\/JVI.74.18.8732-8739.2000","article-title":"Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A\/D junction element","volume":"74","author":"Haberman","year":"2000","journal-title":"J Virol"},{"issue":"12","key":"bib46","doi-asserted-by":"publisher","first-page":"1521","DOI":"10.1002\/humu.24275","article-title":"PRPH2 mutation update: in silico assessment of 245 reported and 7 novel variants in patients with retinal disease","volume":"42","author":"Peeters","year":"2021","journal-title":"Hum Mutat"},{"issue":"11","key":"bib47","doi-asserted-by":"publisher","first-page":"1817","DOI":"10.3390\/genes12111817","article-title":"Genetic and phenotypic landscape of PRPH2-associated retinal dystrophy in Japan","volume":"12","author":"Oishi","year":"2021","journal-title":"Genes"},{"issue":"1","key":"bib48","doi-asserted-by":"publisher","first-page":"11309","DOI":"10.1038\/s41598-019-47721-4","article-title":"Optimized CRISPR\/Cas9-mediated in vivo genome engineering applicable to monitoring dynamics of endogenous proteins in the mouse neural tissues","volume":"9","author":"Matsuda","year":"2019","journal-title":"Sci Rep"},{"key":"bib1","doi-asserted-by":"crossref","DOI":"10.1016\/B978-0-7506-4063-3.50040-9","article-title":"Retinitis pigmentosa","volume-title":"StatPearls","author":"O'Neal"},{"key":"bib25","first-page":"107653","article-title":"Pronuclear Microinjection during S-phase increases the efficiency of CRISPR-Cas9-assisted knockin of large DNA donors in mouse zygotes","volume-title":"Cell Rep","author":"Abe","year":"2020"},{"key":"bib32","doi-asserted-by":"crossref","article-title":"grID: a CRISPR-Cas9 guide RNA database and resource for genome-editing","author":"Jaskula-Ranga","DOI":"10.1101\/097352"}],"container-title":["Investigative Ophthalmology &amp; Visual Science"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/iovs.arvojournals.org\/article.aspx?articleid=2802250","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T21:31:53Z","timestamp":1791927113000},"score":1,"resource":{"primary":{"URL":"https:\/\/iovs.arvojournals.org\/article.aspx?articleid=2802250"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,11,18]]},"references-count":48,"journal-issue":{"issue":"13","published-print":{"date-parts":[[2024,11,4]]}},"URL":"https:\/\/doi.org\/10.1167\/iovs.65.13.38","relation":{},"ISSN":["1552-5783"],"issn-type":[{"value":"1552-5783","type":"print"}],"subject":[],"published":{"date-parts":[[2024,11,18]]}}}