CRISPR applications in ophthalmologic PINION genome surgery

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dc.citation.issue3
dc.citation.volume28
dc.contributor.authorCabral, Thiago [UNIFESP]
dc.contributor.authorDiCarlo, James E.
dc.contributor.authorJustus, Sally
dc.contributor.authorSengillo, Jesse D.
dc.contributor.authorXu, Yu
dc.contributor.authorTsang, Stephen H.
dc.coveragePhiladelphia
dc.date.accessioned2020-07-17T14:02:13Z
dc.date.available2020-07-17T14:02:13Z
dc.date.issued2017
dc.description.abstractPurpose of review The present review seeks to summarize and discuss the application of clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems (Cas) for genome editing, also called genome surgery, in the field of ophthalmology. Recent findings Precision medicine is an emerging approach for disease treatment and prevention that takes into account the variability of an individual's genetic sequence. Various groups have used CRISPR-Cas genome editing to make significant progress in mammalian preclinical models of eye disease, the basic science of eye development in zebrafish, the in vivo modification of ocular tissue, and the correction of stem cells with therapeutic applications. In addition, investigators have creatively used the targeted mutagenic potential of CRISPR-Cas systems to target pathogenic alleles in vitro. Summary Over the past year, CRISPR Cas genome editing has been used to correct pathogenic mutations in vivo and in transplantable stem cells. Although off-target mutagenesis remains a concern, improvement in CRISPRCas technology and careful screening for undesired mutations will likely lead to clinical eye therapeutics employing CRISPR-Cas systems in the near future.en
dc.description.affiliationColumbia Univ, Dept Ophthalmol, Med Ctr, Jonas Childrens Vis Care, 635 West 165th St,5th Floor, New York, NY 10032 USA
dc.description.affiliationColumbia Univ, Dept Ophthalmol, Med Ctr, Bernard & Shirlee Brown Glaucoma Lab, 635 West 165th St,5th Floor, New York, NY 10032 USA
dc.description.affiliationNew York Presbyterian Hosp, Edward S Harkness Eye Inst, 635 West 165th St,5th Floor, New York, NY 10032 USA
dc.description.affiliationUniv Fed Espirito Santo, Dept Ophthalmol, Vitoria, Brazil
dc.description.affiliationUniv Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, Brazil
dc.description.affiliationSuny Downstate Med Ctr, Brooklyn, NY 11203 USA
dc.description.affiliationShanghai Jiao Tong Univ, Dept Ophthalmol, Xin Hua Hosp, Shanghai, Peoples R China
dc.description.affiliationColumbia Univ, Inst Human Nutr, Dept Pathol & Cell Biol, Coll Phys & Surg, New York, NY 10032 USA
dc.description.affiliationUnifespUniv Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipNational Institutes of Health
dc.description.sponsorshipNational Cancer Institute Core
dc.description.sponsorshipResearch to Prevent Blindness (RPB) Physician-Scientist Award
dc.description.sponsorshipRPB, New York, NY, USA
dc.description.sponsorshipICO-Retina Research Foundation Helmerich Fellowships
dc.description.sponsorshipRetina Research Foundation
dc.description.sponsorshipICO Foundation (International Council of Ophthalmology)
dc.description.sponsorshipRPB medical student 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.sponsorshipIDNational Institutes of Health: 5P30EY019007
dc.description.sponsorshipIDNational Institutes of Health: R01EY018213
dc.description.sponsorshipIDNational Institutes of Health: R01EY024698
dc.description.sponsorshipIDNational Institutes of Health: 1R01EY026682
dc.description.sponsorshipIDNational Institutes of Health: R21AG050437
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 Grant: C-NY05-0705-0312
dc.format.extent252-259
dc.identifierhttp://dx.doi.org/10.1097/ICU.0000000000000359
dc.identifier.citationCurrent Opinion In Ophthalmology. Philadelphia, v. 28, n. 3, p. 252-259, 2017.
dc.identifier.doi10.1097/ICU.0000000000000359
dc.identifier.issn1040-8738
dc.identifier.urihttps://repositorio.unifesp.br/handle/11600/54688
dc.identifier.wosWOS:000399390800008
dc.language.isoeng
dc.publisherLippincott Williams & Wilkins
dc.relation.ispartofCurrent Opinion In Ophthalmology
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectadeno-associated virusen
dc.subjectCRISPR-Casen
dc.subjectembryonic stem cellsen
dc.subjectinduced pluripotent stem cellsen
dc.subjectprecision medicineen
dc.titleCRISPR applications in ophthalmologic PINION genome surgeryen
dc.typeinfo:eu-repo/semantics/article
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