Correction of Monogenic and Common Retinal Disorders with Gene Therapy

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dc.citation.issue2
dc.citation.volume8
dc.contributor.authorSengillo, Jesse D.
dc.contributor.authorJustus, Sally
dc.contributor.authorCabral, Thiago [UNIFESP]
dc.contributor.authorTsang, Stephen H.
dc.coverageBasel
dc.date.accessioned2020-07-17T14:03:02Z
dc.date.available2020-07-17T14:03:02Z
dc.date.issued2017
dc.description.abstractThe past decade has seen major advances in gene-based therapies, many of which show promise for translation to human disease. At the forefront of research in this field is ocular disease, as the eye lends itself to gene-based interventions due to its accessibility, relatively immune-privileged status, and ability to be non-invasively monitored. A landmark study in 2001 demonstrating successful gene therapy in a large-animal model for Leber congenital amaurosis set the stage for translation of these strategies from the bench to the bedside. Multiple clinical trials have since initiated for various retinal diseases, and further improvements in gene therapy techniques have engendered optimism for alleviating inherited blinding disorders. This article provides an overview of gene-based strategies for retinal disease, current clinical trials that engage these strategies, and the latest techniques in genome engineering, which could serve as the next frontline of therapeutic interventions.en
dc.description.affiliationColumbia Univ, Med Ctr, Dept Ophthalmol, Jonas Childrens Vis Care, New York, NY 10032 USA
dc.description.affiliationColumbia Univ, Med Ctr, Dept Ophthalmol, Bernard & Shirlee Brown Glaucoma Lab, New York, NY 10032 USA
dc.description.affiliationNew York Presbyterian Hosp, Edward S Harkness Eye Inst, New York, NY 10032 USA
dc.description.affiliationSuny Downstate Med Ctr, Brooklyn, NY 11203 USA
dc.description.affiliationUniv Fed Espirito Santo, Dept Ophthalmol, BR-29075910 Vitoria, Brazil
dc.description.affiliationUniv Fed Sao Paulo, Dept Ophthalmol, BR-04021001 Sao Paulo, Brazil
dc.description.affiliationColumbia Univ Coll Phys & Surg, Inst Human Nutr, Dept Pathol & Cell Biol, 630 W 168th St, New York, NY 10032 USA
dc.description.affiliationUnifespUniv Fed Sao Paulo, Dept Ophthalmol, BR-04021001 Sao Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipNational Institutes of Health
dc.description.sponsorshipNational Cancer Institute
dc.description.sponsorshipResearch to Prevent Blindness (RPB) Physician-Scientist Award
dc.description.sponsorshipRPB, New York, NY, USA
dc.description.sponsorshipRPB Medical Student Research Fellowship
dc.description.sponsorshipInternational Council of Ophthalmology-Retina Research Foundation Helmerich Fellowship
dc.description.sponsorshipTistou and Charlotte Kerstan Foundation
dc.description.sponsorshipSchneeweiss Stem Cell Fund, New York State
dc.description.sponsorshipFoundation Fighting Blindness New York Regional Research Center Grant
dc.description.sponsorshipJoel Hoffman Fund
dc.description.sponsorshipProfessor Gertrude Rothschild Stem Cell Foundation
dc.description.sponsorshipGebroe Family Foundation
dc.description.sponsorshipIDNIH: 5P30EY019007
dc.description.sponsorshipIDNIH: R01EY018213
dc.description.sponsorshipIDNIH: R01EY024698
dc.description.sponsorshipIDNIH: R01EY026682
dc.description.sponsorshipIDNIH: R21AG050437
dc.description.sponsorshipIDINCA: 5P30CA013696
dc.description.sponsorshipIDSchneeweiss Stem Cell Fund, New York State: C029572
dc.description.sponsorshipIDFoundation Fighting Blindness New York Regional Research Center Grant: C-NY05-0705-0312
dc.format.extent-
dc.identifierhttp://dx.doi.org/10.3390/genes8020053
dc.identifier.citationGenes. Basel, v. 8, n. 2, p. -, 2017.
dc.identifier.doi10.3390/genes8020053
dc.identifier.fileWOS000399058000008.pdf
dc.identifier.issn2073-4425
dc.identifier.urihttps://repositorio.unifesp.br/handle/11600/55132
dc.identifier.wosWOS:000399058000008
dc.language.isoeng
dc.publisherMdpi
dc.relation.ispartofGenes
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectgene therapyen
dc.subjectgenome engineeringen
dc.subjectCRISPRen
dc.subjectretinal degenerationen
dc.subjectinherited retinal diseaseen
dc.titleCorrection of Monogenic and Common Retinal Disorders with Gene Therapyen
dc.typeinfo:eu-repo/semantics/article
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