CRISPR-Cas Genome Surgery in Ophthalmology

Página do item simplificado
dc.citation.issue3
dc.citation.volume6
dc.contributor.authorDiCarlo, James E.
dc.contributor.authorSengillo, Jesse D.
dc.contributor.authorJustus, Sally
dc.contributor.authorCabral, Thiago [UNIFESP]
dc.contributor.authorTsang, Stephen H.
dc.contributor.authorMahajan, Vinit B.
dc.coverageRockville
dc.date.accessioned2020-07-13T11:53:16Z
dc.date.available2020-07-13T11:53:16Z
dc.date.issued2017
dc.description.abstractGenetic disease affecting vision can significantly impact patient quality of life. Gene therapy seeks to slow the progression of these diseases by treating the underlying etiology at the level of the genome. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated systems (Cas) represent powerful tools for studying diseases through the creation of model organisms generated by targeted modification and by the correction of disease mutations for therapeutic purposes. CRISPR-Cas systems have been applied successfully to the visual sciences and study of ophthalmic disease -from the modification of zebrafish and mammalian models of eye development and disease, to the correction of pathogenic mutations in patientderived stem cells. Recent advances in CRISPR-Cas delivery and optimization boast improved functionality that continues to enhance genome-engineering applications in the eye. This review provides a synopsis of the recent implementations of CRISPRCas tools in the field of ophthalmology.en
dc.description.affiliationColumbia Univ, Dept Ophthalmol, Jonas Childrens Vis Care, New York, NY 10027 USA
dc.description.affiliationColumbia Univ, Dept Ophthalmol, Bernard & Shirlee Brown Glaucoma Lab, New York, NY 10027 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, Vitoria, Brazil
dc.description.affiliationUniv Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, Brazil
dc.description.affiliationColumbia Univ, Coll Phys & Surg, Inst Human Nutr, Dept Pathol & Cell Biol, New York, NY USA
dc.description.affiliationStanford Univ, Dept Ophthalmol, Byers Eye Inst, Omics Lab, Palo Alto, CA 94304 USA
dc.description.affiliationStanford Univ, Byers Eye Inst, Dept Ophthalmol, Palo Alto, CA 94304 USA
dc.description.affiliationUnifespUniv Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipNational Institutes of Health (NIH
dc.description.sponsorshipBethesda, MD)
dc.description.sponsorshipNIH
dc.description.sponsorshipNational Cancer Institute Core
dc.description.sponsorshipResearch to Prevent Blindness (RPB) Physician-Scientist Award, unrestricted funds from RPB, New York, NY, USA
dc.description.sponsorshipRPB medical student fellowship (JDS)
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
dc.description.sponsorshipGebroe Family Foundation
dc.description.sponsorshipICO-Retina Research Foundation Helmerich Fellowships
dc.description.sponsorshipRetina Research Foundation in cooperation with the ICO Foundation
dc.description.sponsorshipIDNIH: R01EY026682
dc.description.sponsorshipIDNIH: R01EY024665
dc.description.sponsorshipIDNIH: R01EY025225
dc.description.sponsorshipIDNIH: R01EY024698
dc.description.sponsorshipIDNIH: R21AG050437
dc.description.sponsorshipIDNIH: R01EY024698
dc.description.sponsorshipIDNIH: R21AG050437
dc.description.sponsorshipIDNIH: 5P30EY019007
dc.description.sponsorshipIDNIH: R01EY018213
dc.description.sponsorshipIDNIH: \1R01EY026682
dc.description.sponsorshipIDNational Cancer Institute Core: 5P30CA013696
dc.description.sponsorshipIDSchneeweiss Stem Cell Fund, New York State: C029572
dc.description.sponsorshipIDFoundation Fighting Blindness New York Regional Research Center: C-NY05-0705-0312
dc.format.extent-
dc.identifierhttp://dx.doi.org/10.1167/tvst.6.3.13
dc.identifier.citationTranslational Vision Science & Technology. Rockville, v. 6, n. 3, p. -, 2017.
dc.identifier.doi10.1167/tvst.6.3.13
dc.identifier.fileWOS000410957800013.pdf
dc.identifier.issn2164-2591
dc.identifier.urihttps://repositorio.unifesp.br/handle/11600/54498
dc.identifier.wosWOS:000410957800013
dc.language.isoeng
dc.publisherAssoc Research Vision Ophthalmology Inc
dc.relation.ispartofTranslational Vision Science & Technology
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectCRISPR-Casen
dc.subjectgenome engineeringen
dc.subjectgene therapyen
dc.subjectsynthetic biologyen
dc.subjectprecision medicineen
dc.titleCRISPR-Cas Genome Surgery in Ophthalmologyen
dc.typeinfo:eu-repo/semantics/article
Arquivos
Coleções
EPM - Artigos