{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,6]],"date-time":"2026-07-06T00:12:32Z","timestamp":1783296752753,"version":"3.54.6"},"reference-count":63,"publisher":"Elsevier BV","issue":"12","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,10,7]],"date-time":"2024-10-07T00:00:00Z","timestamp":1728259200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004431","name":"Foundation of France","doi-asserted-by":"publisher","award":["00012185"],"award-info":[{"award-number":["00012185"]}],"id":[{"id":"10.13039\/501100004431","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100013875","name":"Retina France","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100013875","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007393","name":"Association Fran\u00e7aise contre les Myopathies","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007393","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004923","name":"AFM-T\u00e9l\u00e9thon","doi-asserted-by":"publisher","award":["16599"],"award-info":[{"award-number":["16599"]}],"id":[{"id":"10.13039\/501100004923","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002915","name":"Foundation for Medical Research","doi-asserted-by":"publisher","award":["VL\/DLS\/GP\/vk111114"],"award-info":[{"award-number":["VL\/DLS\/GP\/vk111114"]}],"id":[{"id":"10.13039\/501100002915","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2024,12]]},"DOI":"10.1016\/j.ymthe.2024.10.004","type":"journal-article","created":{"date-parts":[[2024,10,9]],"date-time":"2024-10-09T15:14:55Z","timestamp":1728486895000},"page":"4319-4336","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["Dual CRALBP isoforms unveiled: iPSC-derived retinal modeling and AAV2\/5-RLBP1 gene transfer raise considerations for effective therapy"],"prefix":"10.1016","volume":"32","author":[{"given":"Krishna","family":"Damodar","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregor","family":"Dubois","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Guillou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daria","family":"Mamaeva","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie","family":"Pequignot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nejla","family":"Erkilic","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Sanjurjo-Soriano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hassan","family":"Boukhaddaoui","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Florence","family":"Bernex","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B\u00e9atrice","family":"Bocquet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00e9r\u00f4me","family":"Vialaret","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yvan","family":"Arsenijevic","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. Michael","family":"Redmond","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Hirtz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Meunier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Brabet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9626-8807","authenticated-orcid":false,"given":"Vasiliki","family":"Kalatzis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2024.10.004_bib1","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1016\/bs.pmbts.2015.06.001","article-title":"Introduction to the Retina","volume":"134","author":"Grossniklaus","year":"2015","journal-title":"Prog. Mol. Biol. Transl. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib2","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1016\/j.preteyeres.2010.03.004","article-title":"The molecular basis of human retinal and vitreoretinal diseases","volume":"29","author":"Berger","year":"2010","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.ymthe.2024.10.004_bib3","doi-asserted-by":"crossref","first-page":"4719","DOI":"10.1167\/iovs.05-1488","article-title":"Homozygous deletion related to Alu repeats in RLBP1 causes retinitis punctata albescens","volume":"47","author":"Humbert","year":"2006","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib4","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/ng1097-198","article-title":"Mutation of the gene encoding cellular retinaldehyde-binding protein in autosomal recessive retinitis pigmentosa","volume":"17","author":"Maw","year":"1997","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2024.10.004_bib5","first-page":"1000","article-title":"Recessive mutations in the RLBP1 gene encoding cellular retinaldehyde-binding protein in a form of retinitis punctata albescens","volume":"40","author":"Morimura","year":"1999","journal-title":"Invest Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib6","doi-asserted-by":"crossref","DOI":"10.1016\/j.xops.2021.100052","article-title":"Retinitis Punctata Albescens and RLBP1-Allied Phenotypes: Phenotype-Genotype Correlation and Natural History in the Aim of Gene Therapy","volume":"1","author":"Bocquet","year":"2021","journal-title":"Ophthalmol. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib7","first-page":"260","article-title":"Ocular phenotype of bothnia dystrophy, an autosomal recessive retinitis pigmentosa associated with an R234W mutation in the RLBP1 gene","volume":"119","author":"Burstedt","year":"2001","journal-title":"Arch. Ophthalmol."},{"key":"10.1016\/j.ymthe.2024.10.004_bib8","first-page":"995","article-title":"Bothnia dystrophy caused by mutations in the cellular retinaldehyde-binding protein gene (RLBP1) on chromosome 15q26","volume":"40","author":"Burstedt","year":"1999","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib9","doi-asserted-by":"crossref","first-page":"955","DOI":"10.1086\/339688","article-title":"Newfoundland rod-cone dystrophy, an early-onset retinal dystrophy, is caused by splice-junction mutations in RLBP1","volume":"70","author":"Eichers","year":"2002","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2024.10.004_bib10","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1034\/j.1399-0004.2001.590607.x","article-title":"Fundus albipunctatus and retinitis punctata albescens in a pedigree with an R150Q mutation in RLBP1","volume":"59","author":"Katsanis","year":"2001","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymthe.2024.10.004_bib11","first-page":"337","article-title":"Biochemistry of visual pigment regeneration: the Friedenwald lecture","volume":"41","author":"Saari","year":"2000","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib12","doi-asserted-by":"crossref","first-page":"19629","DOI":"10.1073\/pnas.2008211117","article-title":"Shedding new light on the generation of the visual chromophore","volume":"117","author":"Palczewski","year":"2020","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.10.004_bib13","doi-asserted-by":"crossref","first-page":"449","DOI":"10.1016\/j.cell.2005.06.042","article-title":"Rpe65 is the retinoid isomerase in bovine retinal pigment epithelium","volume":"122","author":"Jin","year":"2005","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2024.10.004_bib14","doi-asserted-by":"crossref","first-page":"12413","DOI":"10.1073\/pnas.0503460102","article-title":"RPE65 is the isomerohydrolase in the retinoid visual cycle","volume":"102","author":"Moiseyev","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.10.004_bib15","doi-asserted-by":"crossref","first-page":"13658","DOI":"10.1073\/pnas.0504167102","article-title":"Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle","volume":"102","author":"Redmond","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.10.004_bib16","doi-asserted-by":"crossref","first-page":"739","DOI":"10.1016\/S0896-6273(01)00248-3","article-title":"Visual cycle impairment in cellular retinaldehyde binding protein (CRALBP) knockout mice results in delayed dark adaptation","volume":"29","author":"Saari","year":"2001","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2024.10.004_bib17","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/S0896-6273(02)00912-1","article-title":"Isomerization and oxidation of vitamin a in cone-dominant retinas: a novel pathway for visual-pigment regeneration in daylight","volume":"36","author":"Mata","year":"2002","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2024.10.004_bib18","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1038\/nn.2258","article-title":"Intra-retinal visual cycle required for rapid and complete cone dark adaptation","volume":"12","author":"Wang","year":"2009","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib19","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2023.112982","article-title":"Rapid RGR-dependent visual pigment recycling is mediated by the RPE and specialized Muller glia","volume":"42","author":"Tworak","year":"2023","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2024.10.004_bib20","doi-asserted-by":"crossref","first-page":"727","DOI":"10.1172\/JCI79651","article-title":"CRALBP supports the mammalian retinal visual cycle and cone vision","volume":"125","author":"Xue","year":"2015","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2024.10.004_bib21","doi-asserted-by":"crossref","DOI":"10.21037\/aes-20-81","article-title":"Retinal imaging in inherited retinal diseases","volume":"5","author":"Georgiou","year":"2020","journal-title":"Ann. Eye Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib22","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.ymthe.2024.10.004_bib23","doi-asserted-by":"crossref","first-page":"344","DOI":"10.1038\/3813","article-title":"Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle","volume":"20","author":"Redmond","year":"1998","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2024.10.004_bib24","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2015.22","article-title":"AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout mice","volume":"2","author":"Choi","year":"2015","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2024.10.004_bib25","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2014.11","article-title":"Proof of concept for AAV2\/5-mediated gene therapy in iPSc-derived retinal pigment epithelium of choroideremia patients","volume":"1","author":"Cereso","year":"2014","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2024.10.004_bib26","doi-asserted-by":"crossref","first-page":"3573","DOI":"10.1093\/hmg\/ddx244","article-title":"Pathogenicity of a novel missense variant associated with choroideremia and its impact on gene replacement therapy","volume":"26","author":"Torriano","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2024.10.004_bib27","doi-asserted-by":"crossref","first-page":"662","DOI":"10.1038\/nprot.2009.33","article-title":"A protocol for the culture and differentiation of highly polarized human retinal pigment epithelial cells","volume":"4","author":"Sonoda","year":"2009","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.ymthe.2024.10.004_bib28","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.169426","article-title":"TBC1D32 variants disrupt retinal ciliogenesis and cause retinitis pigmentosa","volume":"8","author":"Bocquet","year":"2023","journal-title":"JCI Insight"},{"key":"10.1016\/j.ymthe.2024.10.004_bib29","doi-asserted-by":"crossref","first-page":"3106","DOI":"10.1021\/bi00176a045","article-title":"Control of substrate flow at a branch in the visual cycle","volume":"33","author":"Saari","year":"1994","journal-title":"Biochemistry"},{"key":"10.1016\/j.ymthe.2024.10.004_bib30","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1083\/jcb.200405110","article-title":"Retinosomes: new insights into intracellular managing of hydrophobic substances in lipid bodies","volume":"166","author":"Imanishi","year":"2004","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2024.10.004_bib31","doi-asserted-by":"crossref","first-page":"1444","DOI":"10.1038\/nm.2260","article-title":"Noninvasive multiphoton fluorescence microscopy resolves retinol and retinal condensation products in mouse eyes","volume":"16","author":"Palczewska","year":"2010","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2024.10.004_bib32","doi-asserted-by":"crossref","DOI":"10.1096\/fj.202002754R","article-title":"Novel roles for voltage-gated T-type Ca2+ and ClC-2 channels in phagocytosis and angiogenic factor balance identified in human iPSC-derived RPE","volume":"35","author":"Mamaeva","year":"2021","journal-title":"FASEB J."},{"key":"10.1016\/j.ymthe.2024.10.004_bib33","doi-asserted-by":"crossref","first-page":"18759","DOI":"10.1074\/jbc.M109.064329","article-title":"FATP1 inhibits 11-cis retinol formation via interaction with the visual cycle retinoid isomerase RPE65 and lecithin:retinol acyltransferase","volume":"285","author":"Guignard","year":"2010","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2024.10.004_bib34","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1111\/ceo.13917","article-title":"Inherited retinal diseases: Therapeutics, clinical trials and end points-A review","volume":"49","author":"Georgiou","year":"2021","journal-title":"Clin. Exp. Ophthalmol."},{"key":"10.1016\/j.ymthe.2024.10.004_bib35","doi-asserted-by":"crossref","first-page":"703","DOI":"10.1083\/jcb.97.3.703","article-title":"Immunocytochemical localization of two retinoid-binding proteins in vertebrate retina","volume":"97","author":"Bunt-Milam","year":"1983","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.ymthe.2024.10.004_bib36","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1523\/JNEUROSCI.21-01-00053.2001","article-title":"The Rpe65 Leu450Met variation increases retinal resistance against light-induced degeneration by slowing rhodopsin regeneration","volume":"21","author":"Wenzel","year":"2001","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib37","doi-asserted-by":"crossref","first-page":"972","DOI":"10.1046\/j.1471-4159.2003.01608.x","article-title":"Differentiation of human retinal pigment epithelial cells into neuronal phenotype by N-(4-hydroxyphenyl)retinamide","volume":"84","author":"Chen","year":"2003","journal-title":"J.\u00a0Neurochem."},{"key":"10.1016\/j.ymthe.2024.10.004_bib38","doi-asserted-by":"crossref","first-page":"5089","DOI":"10.1167\/iovs.09-3797","article-title":"The 11-cis-retinol dehydrogenase activity of RDH10 and its interaction with visual cycle proteins","volume":"50","author":"Farjo","year":"2009","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib39","article-title":"Microphthalmia-associated transcription factor regulates the visual cycle genes Rlbp1 and Rdh5 in the retinal pigment epithelium","volume":"6","author":"Wen","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2024.10.004_bib40","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.redox.2017.05.015","article-title":"Altered bioenergetics and enhanced resistance to oxidative stress in human retinal pigment epithelial cells from donors with age-related macular degeneration","volume":"13","author":"Ferrington","year":"2017","journal-title":"Redox Biol."},{"key":"10.1016\/j.ymthe.2024.10.004_bib41","doi-asserted-by":"crossref","first-page":"29874","DOI":"10.1074\/jbc.M503603200","article-title":"The retinal G\u00a0protein-coupled receptor (RGR) enhances isomerohydrolase activity independent of light","volume":"280","author":"Wenzel","year":"2005","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2024.10.004_bib42","doi-asserted-by":"crossref","first-page":"3812","DOI":"10.1167\/iovs.08-1957","article-title":"Duplication and divergence of zebrafish CRALBP genes uncovers novel role for RPE- and Muller-CRALBP in cone vision","volume":"49","author":"Collery","year":"2008","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib43","doi-asserted-by":"crossref","first-page":"746","DOI":"10.1002\/pro.5560070324","article-title":"Structural and functional characterization of recombinant human cellular retinaldehyde-binding protein","volume":"7","author":"Crabb","year":"1998","journal-title":"Protein Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib44","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1016\/j.exer.2005.06.015","article-title":"Focus on molecules: cellular retinaldehyde-binding protein (CRALBP)","volume":"81","author":"Saari","year":"2005","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.ymthe.2024.10.004_bib45","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1016\/j.exer.2004.04.001","article-title":"Support for a proposed retinoid-processing protein complex in apical retinal pigment epithelium","volume":"79","author":"Bonilha","year":"2004","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.ymthe.2024.10.004_bib46","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1167\/iovs.03-0989","article-title":"Cellular retinaldehyde-binding protein interacts with ERM-binding phosphoprotein 50 in retinal pigment epithelium","volume":"45","author":"Nawrot","year":"2004","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib47","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2024.114143","article-title":"Dominant role for pigment epithelial CRALBP in supplying visual chromophore to photoreceptors","volume":"43","author":"Bassetto","year":"2024","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2024.10.004_bib48","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.71473","article-title":"Disturbed retinoid metabolism upon loss of rlbp1a impairs cone function and leads to subretinal lipid deposits and photoreceptor degeneration in the zebrafish retina","volume":"10","author":"Schlegel","year":"2021","journal-title":"Elife"},{"key":"10.1016\/j.ymthe.2024.10.004_bib49","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for Leber\u2019s congenital amaurosis","volume":"358","author":"Maguire","year":"2008","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymthe.2024.10.004_bib50","doi-asserted-by":"crossref","first-page":"2231","DOI":"10.1056\/NEJMoa0802268","article-title":"Effect of gene therapy on visual function in Leber\u2019s congenital amaurosis","volume":"358","author":"Bainbridge","year":"2008","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymthe.2024.10.004_bib51","doi-asserted-by":"crossref","first-page":"1076","DOI":"10.1016\/j.ymthe.2017.03.008","article-title":"Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward","volume":"25","author":"Bennett","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.10.004_bib52","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1016\/S0014-4835(02)00308-1","article-title":"CRALBP transcriptional regulation in ciliary epithelial, retinal Muller and retinal pigment epithelial cells","volume":"76","author":"Kennedy","year":"2003","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.ymthe.2024.10.004_bib53","doi-asserted-by":"crossref","first-page":"6767","DOI":"10.1074\/jbc.RA120.012695","article-title":"Effects of deficiency in the RLBP1-encoded visual cycle protein CRALBP on visual dysfunction in humans and mice","volume":"295","author":"Lima de Carvalho","year":"2020","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2024.10.004_bib54","first-page":"223","article-title":"Localizations of visual cycle components in retinal pigment epithelium","volume":"15","author":"Huang","year":"2009","journal-title":"Mol. Vis."},{"key":"10.1016\/j.ymthe.2024.10.004_bib55","doi-asserted-by":"crossref","first-page":"12397","DOI":"10.1074\/jbc.M207300200","article-title":"Disease-causing mutations in the cellular retinaldehyde binding protein tighten and abolish ligand interactions","volume":"278","author":"Golovleva","year":"2003","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2024.10.004_bib56","doi-asserted-by":"crossref","first-page":"18545","DOI":"10.1073\/pnas.0907454106","article-title":"Bothnia dystrophy is caused by domino-like rearrangements in cellular retinaldehyde-binding protein mutant R234W","volume":"106","author":"He","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2024.10.004_bib57","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1186\/s13287-022-03146-x","article-title":"Retinoic acid delays initial photoreceptor differentiation and results in a highly structured mature retinal organoid","volume":"13","author":"Sanjurjo-Soriano","year":"2022","journal-title":"Stem Cell Res. Ther."},{"key":"10.1016\/j.ymthe.2024.10.004_bib58","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1016\/j.ebiom.2018.12.010","article-title":"Zika virus induces strong inflammatory responses and impairs homeostasis and function of the human retinal pigment epithelium","volume":"39","author":"Simonin","year":"2019","journal-title":"EBioMedicine"},{"key":"10.1016\/j.ymthe.2024.10.004_bib59","doi-asserted-by":"crossref","first-page":"8258","DOI":"10.1021\/acsomega.1c04770","article-title":"Deciphering Black Extrinsic Tooth Stain Composition in Children Using Metaproteomics","volume":"7","author":"Hirtz","year":"2022","journal-title":"ACS Omega"},{"key":"10.1016\/j.ymthe.2024.10.004_bib60","doi-asserted-by":"crossref","first-page":"W521","DOI":"10.1093\/nar\/gki380","article-title":"Assembly PCR oligo maker: a tool for designing oligodeoxynucleotides for constructing long DNA molecules for RNA production","volume":"33","author":"Rydzanicz","year":"2005","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2024.10.004_bib61","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1167\/iovs.13-11740","article-title":"Retinoid uptake, processing, and secretion in human iPS-RPE support the visual cycle","volume":"55","author":"Muniz","year":"2014","journal-title":"Invest Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2024.10.004_bib62","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1016\/0042-6989(86)90185-9","article-title":"A simple procedure for the extraction of the native chromophore of visual pigments: the formaldehyde method","volume":"26","author":"Suzuki","year":"1986","journal-title":"Vis. Res."},{"key":"10.1016\/j.ymthe.2024.10.004_bib63","doi-asserted-by":"crossref","first-page":"174","DOI":"10.1159\/000321118","article-title":"Simple and efficient: validation of a cotton wick electrode for animal electroretinography","volume":"45","author":"Chekroud","year":"2011","journal-title":"Ophthalmic Res."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001624006646?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001624006646?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,7]],"date-time":"2025-11-07T20:41:23Z","timestamp":1762548083000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001624006646"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12]]},"references-count":63,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["S1525001624006646"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2024.10.004","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2024.04.24.590768","asserted-by":"object"}]},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2024,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Dual CRALBP isoforms unveiled: iPSC-derived retinal modeling and AAV2\/5-RLBP1 gene transfer raise considerations for effective therapy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2024.10.004","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}