{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T07:51:35Z","timestamp":1793173895993,"version":"4.5.0"},"reference-count":58,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2020,3,1]],"date-time":"2020-03-01T00:00:00Z","timestamp":1583020800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,3,1]],"date-time":"2020-03-01T00:00:00Z","timestamp":1583020800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,1,8]],"date-time":"2020-01-08T00:00:00Z","timestamp":1578441600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000305","name":"National Eye Research Centre","doi-asserted-by":"publisher","award":["BRI 019"],"award-info":[{"award-number":["BRI 019"]}],"id":[{"id":"10.13039\/501100000305","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Frost Charitable Trust"},{"name":"Above & Beyond"},{"name":"Bristol"},{"DOI":"10.13039\/100002089","name":"Fight for Sight","doi-asserted-by":"publisher","award":["1730\/1731"],"award-info":[{"award-number":["1730\/1731"]}],"id":[{"id":"10.13039\/100002089","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Elizabeth Blackwell Institute for Health Research"},{"DOI":"10.13039\/501100000883","name":"University of Bristol","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000883","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"Medical Research Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"National Institute for Health Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014461","name":"Biomedical Research Centre","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014461","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012617","name":"Moorfields Eye Hospital NHS Foundation Trust","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100012617","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001285","name":"UCL Institute of Ophthalmology","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001285","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":[[2020,3]]},"DOI":"10.1016\/j.ymthe.2019.12.012","type":"journal-article","created":{"date-parts":[[2020,1,9]],"date-time":"2020-01-09T20:28:51Z","timestamp":1578601731000},"page":"820-829","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":97,"title":["Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9"],"prefix":"10.1016","volume":"28","author":[{"given":"Jiahui","family":"Wu","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oliver H.","family":"Bell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David A.","family":"Copland","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alison","family":"Young","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John R.","family":"Pooley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryea","family":"Maswood","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel S.","family":"Evans","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peng Tee","family":"Khaw","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robin R.","family":"Ali","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew D.","family":"Dick","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Colin J.","family":"Chu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2019.12.012_bib1","doi-asserted-by":"crossref","first-page":"2081","DOI":"10.1016\/j.ophtha.2014.05.013","article-title":"Global prevalence of glaucoma and projections of glaucoma burden through 2040: a systematic review and meta-analysis","volume":"121","author":"Tham","year":"2014","journal-title":"Ophthalmology"},{"key":"10.1016\/j.ymthe.2019.12.012_bib2","doi-asserted-by":"crossref","first-page":"1295","DOI":"10.1016\/S0140-6736(14)62111-5","article-title":"Latanoprost for open-angle glaucoma (UKGTS): a randomised, multicentre, placebo-controlled trial","volume":"385","author":"Garway-Heath","year":"2015","journal-title":"Lancet"},{"key":"10.1016\/j.ymthe.2019.12.012_bib3","doi-asserted-by":"crossref","first-page":"498","DOI":"10.1016\/S0002-9394(98)00272-4","article-title":"The effectiveness of intraocular pressure reduction in the treatment of normal-tension glaucoma","volume":"126","year":"1998","journal-title":"Am. J. Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib4","doi-asserted-by":"crossref","first-page":"1711","DOI":"10.1016\/S0140-6736(04)16257-0","article-title":"Primary open-angle glaucoma","volume":"363","author":"Weinreb","year":"2004","journal-title":"Lancet"},{"key":"10.1016\/j.ymthe.2019.12.012_bib5","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1097\/IJG.0000000000000389","article-title":"A Systematic Review of End-of-Life Visual Impairment in Open-Angle Glaucoma: An Epidemiological Autopsy","volume":"25","author":"Mokhles","year":"2016","journal-title":"J.\u00a0Glaucoma"},{"key":"10.1016\/j.ymthe.2019.12.012_bib6","doi-asserted-by":"crossref","first-page":"598","DOI":"10.1016\/j.ajo.2005.04.051","article-title":"Persistence and adherence with topical glaucoma therapy","volume":"140","author":"Nordstrom","year":"2005","journal-title":"Am. J. Ophthalmol."},{"issue":"Suppl 1","key":"10.1016\/j.ymthe.2019.12.012_bib7","doi-asserted-by":"crossref","first-page":"S57","DOI":"10.1016\/j.survophthal.2008.08.002","article-title":"Adherence and persistence with glaucoma therapy","volume":"53","author":"Schwartz","year":"2008","journal-title":"Surv. Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib8","doi-asserted-by":"crossref","first-page":"2532","DOI":"10.1016\/j.ophtha.2013.07.049","article-title":"Trabeculectomy in the 21st century: a multicenter analysis","volume":"120","author":"Kirwan","year":"2013","journal-title":"Ophthalmology"},{"key":"10.1016\/j.ymthe.2019.12.012_bib9","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1016\/j.ophtha.2011.09.043","article-title":"A twenty-year follow-up study of trabeculectomy: risk factors and outcomes","volume":"119","author":"Landers","year":"2012","journal-title":"Ophthalmology"},{"key":"10.1016\/j.ymthe.2019.12.012_bib10","doi-asserted-by":"crossref","first-page":"804","DOI":"10.1016\/j.ajo.2011.10.024","article-title":"Postoperative complications in the Tube Versus Trabeculectomy (TVT) study during five years of follow-up","volume":"153","author":"Gedde","year":"2012","journal-title":"Am. J. Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib11","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1016\/j.ejpb.2015.04.029","article-title":"The aqueous humor outflow pathways in glaucoma: A unifying concept of disease mechanisms and causative treatment","volume":"95","author":"Braunger","year":"2015","journal-title":"Eur. J. Pharm. Biopharm."},{"key":"10.1016\/j.ymthe.2019.12.012_bib12","doi-asserted-by":"crossref","first-page":"1180","DOI":"10.1136\/bjo.2008.145565","article-title":"Transscleral diode laser cycloablation in patients with good vision","volume":"94","author":"Rotchford","year":"2010","journal-title":"Br. J. Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib13","doi-asserted-by":"crossref","first-page":"1252","DOI":"10.1136\/bjo.87.10.1252","article-title":"A two centre study of the dose-response relation for transscleral diode laser cyclophotocoagulation in refractory glaucoma","volume":"87","author":"Murphy","year":"2003","journal-title":"Br. J. Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib14","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1016\/j.preteyeres.2007.01.002","article-title":"New perspectives in aqueous humor secretion and in glaucoma: the ciliary body as a multifunctional neuroendocrine gland","volume":"26","author":"Coca-Prados","year":"2007","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.ymthe.2019.12.012_bib15","doi-asserted-by":"crossref","first-page":"438","DOI":"10.1038\/gt.2016.14","article-title":"Inducible scAAV2.GRE.MMP1 lowers IOP long-term in a large animal model for steroid-induced glaucoma gene therapy","volume":"23","author":"Borr\u00e1s","year":"2016","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2019.12.012_bib16","doi-asserted-by":"crossref","first-page":"1230","DOI":"10.1093\/hmg\/ddx028","article-title":"Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma","volume":"26","author":"O\u2019Callaghan","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2019.12.012_bib17","doi-asserted-by":"crossref","first-page":"1007","DOI":"10.1038\/s41419-018-1041-8","article-title":"Neuroprotection of retinal ganglion cells by a novel gene therapy construct that achieves sustained enhancement of brain-derived neurotrophic factor\/tropomyosin-related kinase receptor-B signaling","volume":"9","author":"Osborne","year":"2018","journal-title":"Cell Death Dis."},{"key":"10.1016\/j.ymthe.2019.12.012_bib18","doi-asserted-by":"crossref","first-page":"4357","DOI":"10.1167\/iovs.02-1332","article-title":"Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model","volume":"44","author":"Martin","year":"2003","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib19","doi-asserted-by":"crossref","first-page":"11199","DOI":"10.1073\/pnas.1706193114","article-title":"CRISPR-Cas9-based treatment of myocilin-associated glaucoma","volume":"114","author":"Jain","year":"2017","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.12.012_bib20","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1038\/s41588-018-0126-8","article-title":"Genome-wide analyses identify 68 new loci associated with intraocular pressure and improve risk prediction for primary open-angle glaucoma","volume":"50","author":"Khawaja","year":"2018","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2019.12.012_bib21","doi-asserted-by":"crossref","first-page":"28886","DOI":"10.3390\/ijms161226135","article-title":"An Updated Review on the Genetics of Primary Open Angle Glaucoma","volume":"16","author":"Abu-Amero","year":"2015","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib22","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1038\/nature14299","article-title":"In\u00a0vivo genome editing using Staphylococcus aureus Cas9","volume":"520","author":"Ran","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2019.12.012_bib23","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"},{"key":"10.1016\/j.ymthe.2019.12.012_bib24","doi-asserted-by":"crossref","first-page":"F13","DOI":"10.1152\/ajprenal.2000.278.1.F13","article-title":"Structure and function of aquaporin water channels","volume":"278","author":"Verkman","year":"2000","journal-title":"Am. J. Physiol. Renal Physiol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib25","doi-asserted-by":"crossref","first-page":"1513","DOI":"10.1016\/j.bbagen.2013.10.037","article-title":"Aquaporins in the eye: expression, function, and roles in ocular disease","volume":"1840","author":"Schey","year":"2014","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.ymthe.2019.12.012_bib26","doi-asserted-by":"crossref","first-page":"561","DOI":"10.1085\/jgp.20028597","article-title":"Aquaporin deletion in mice reduces intraocular pressure and aqueous fluid production","volume":"119","author":"Zhang","year":"2002","journal-title":"J.\u00a0Gen. Physiol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib27","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.ymthe.2019.12.012_bib28","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1186\/s13059-017-1164-8","article-title":"Efficient precise knockin with a double cut HDR donor after CRISPR\/Cas9-mediated double-stranded DNA cleavage","volume":"18","author":"Zhang","year":"2017","journal-title":"Genome Biol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib29","doi-asserted-by":"crossref","first-page":"1398","DOI":"10.1073\/pnas.98.4.1398","article-title":"Visualization of a water-selective pore by electron crystallography in vitreous ice","volume":"98","author":"Ren","year":"2001","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2019.12.012_bib30","doi-asserted-by":"crossref","first-page":"1473","DOI":"10.1093\/bioinformatics\/btu048","article-title":"Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases","volume":"30","author":"Bae","year":"2014","journal-title":"Bioinformatics"},{"key":"10.1016\/j.ymthe.2019.12.012_bib31","doi-asserted-by":"crossref","first-page":"1268","DOI":"10.1001\/archopht.120.10.1268","article-title":"Reduction of intraocular pressure and glaucoma progression: results from the Early Manifest Glaucoma Trial","volume":"120","author":"Heijl","year":"2002","journal-title":"Arch Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib32","first-page":"1760","article-title":"Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential","volume":"14","author":"Li","year":"2008","journal-title":"Mol. Vis."},{"key":"10.1016\/j.ymthe.2019.12.012_bib33","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1038\/gt.2014.115","article-title":"Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates","volume":"22","author":"Kotterman","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2019.12.012_bib34","doi-asserted-by":"crossref","first-page":"247","DOI":"10.3109\/02713688809047029","article-title":"Cell turnover in ciliary epithelium compared to other slow renewing epithelia in the adult mouse","volume":"7","author":"McDonald","year":"1988","journal-title":"Curr. Eye Res."},{"key":"10.1016\/j.ymthe.2019.12.012_bib35","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.ymthe.2018.10.009","article-title":"Long-Term Effects of In\u00a0Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus","volume":"27","author":"Jo","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.12.012_bib36","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1016\/j.ajpath.2016.12.003","article-title":"Dexamethasone-Induced Ocular Hypertension in Mice: Effects of Myocilin and Route of Administration","volume":"187","author":"Patel","year":"2017","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib37","doi-asserted-by":"crossref","first-page":"e0183142","DOI":"10.1371\/journal.pone.0183142","article-title":"Minimally-invasive glaucoma surgeries (MIGS) for open angle glaucoma: A systematic review and meta-analysis","volume":"12","author":"Lavia","year":"2017","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymthe.2019.12.012_bib38","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1016\/j.jcjo.2016.07.013","article-title":"Glaucoma treatment trends: a review","volume":"52","author":"Conlon","year":"2017","journal-title":"Can. J. Ophthalmol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib39","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1016\/j.preteyeres.2008.04.001","article-title":"Functions of aquaporins in the eye","volume":"27","author":"Verkman","year":"2008","journal-title":"Prog. Retin. Eye Res."},{"key":"10.1016\/j.ymthe.2019.12.012_bib40","doi-asserted-by":"crossref","first-page":"4423","DOI":"10.1167\/iovs.04-0816","article-title":"Aquaporin-dependent water permeation at the mouse ocular surface: in\u00a0vivo microfluorimetric measurements in cornea and conjunctiva","volume":"45","author":"Levin","year":"2004","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib41","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1038\/gt.2010.157","article-title":"Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver","volume":"18","author":"Qiao","year":"2011","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2019.12.012_bib42","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In\u00a0vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2019.12.012_bib43","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1016\/S0140-6736(17)30979-0","article-title":"Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial","volume":"390","author":"Heier","year":"2017","journal-title":"Lancet"},{"key":"10.1016\/j.ymthe.2019.12.012_bib44","doi-asserted-by":"crossref","first-page":"2282","DOI":"10.1016\/j.ymthe.2018.05.025","article-title":"Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I\/IIa Trial by Intravitreal Delivery","volume":"26","author":"Cukras","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2019.12.012_bib45","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1007\/978-1-4939-7522-8_17","article-title":"Screening for Neutralizing Antibodies Against Natural and Engineered AAV Capsids in Nonhuman Primate Retinas","volume":"1715","author":"Day","year":"2018","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib46","doi-asserted-by":"crossref","first-page":"2185","DOI":"10.1167\/iovs.08-2962","article-title":"Depression of intraocular pressure following inactivation of connexin43 in the nonpigmented epithelium of the ciliary body","volume":"50","author":"Calera","year":"2009","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib47","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements","volume":"36","author":"Kosicki","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib48","doi-asserted-by":"crossref","DOI":"10.2741\/1603","article-title":"The induction of inflammation by adenovirus vectors used for gene therapy","volume":"10","author":"Cotter","year":"2005","journal-title":"Front. Biosci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib49","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1097\/01.ijg.0000169409.01635.bc","article-title":"Primate glaucoma models","volume":"14","author":"Rasmussen","year":"2005","journal-title":"J.\u00a0Glaucoma"},{"key":"10.1016\/j.ymthe.2019.12.012_bib50","doi-asserted-by":"crossref","first-page":"6115","DOI":"10.1167\/iovs.12-9570","article-title":"A novel Schlemm\u2019s Canal scaffold increases outflow facility in a human anterior segment perfusion model","volume":"53","author":"Camras","year":"2012","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib51","first-page":"e53731","article-title":"A Magnetic Microbead Occlusion Model to Induce Ocular Hypertension-Dependent Glaucoma in Mice","volume":"109","author":"Ito","year":"2016","journal-title":"J.\u00a0Vis. Exp."},{"key":"10.1016\/j.ymthe.2019.12.012_bib52","doi-asserted-by":"crossref","first-page":"3699","DOI":"10.1167\/iovs.07-1522","article-title":"Characterization of a spontaneous mouse retinal pigment epithelial cell line B6-RPE07","volume":"49","author":"Chen","year":"2008","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.ymthe.2019.12.012_bib53","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1038\/nbt.3437","article-title":"Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9","volume":"34","author":"Doench","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2019.12.012_bib54","doi-asserted-by":"crossref","first-page":"1124","DOI":"10.1089\/hum.2018.027","article-title":"Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells","volume":"29","author":"Gonzalez-Cordero","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2019.12.012_bib55","doi-asserted-by":"crossref","first-page":"2281","DOI":"10.1038\/nprot.2013.143","article-title":"Genome engineering using the CRISPR-Cas9 system","volume":"8","author":"Ran","year":"2013","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.ymthe.2019.12.012_bib56","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1006\/meth.2001.1262","article-title":"Analysis of relative gene expression data using real-time quantitative PCR and the 2(-Delta Delta C(T)) Method","volume":"25","author":"Livak","year":"2001","journal-title":"Methods"},{"key":"10.1016\/j.ymthe.2019.12.012_bib57","doi-asserted-by":"crossref","first-page":"e63002","DOI":"10.1371\/journal.pone.0063002","article-title":"Assessment and in\u00a0vivo scoring of murine experimental autoimmune uveoretinitis using optical coherence tomography","volume":"8","author":"Chu","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymthe.2019.12.012_bib58","doi-asserted-by":"crossref","first-page":"277","DOI":"10.3109\/02713689108996333","article-title":"Human ciliary body in organ culture","volume":"10","author":"Eichhorn","year":"1991","journal-title":"Curr. Eye Res."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001620300071?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001620300071?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,9,30]],"date-time":"2024-09-30T12:11:33Z","timestamp":1727698293000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001620300071"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,3]]},"references-count":58,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2020,3]]}},"alternative-id":["S1525001620300071"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.12.012","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2020,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2019.12.012","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2020.02.011","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 The Author(s).","name":"copyright","label":"Copyright"}]}}