CRISPR applications in ophthalmologic PINION genome surgery

Detalhes bibliográficos
Autor(a) principal: Cabral, Thiago [UNIFESP]
Data de Publicação: 2017
Outros Autores: DiCarlo, James E., Justus, Sally, Sengillo, Jesse D., Xu, Yu, Tsang, Stephen H.
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UNIFESP
dARK ID: ark:/48912/001300000z5s3
Texto Completo: http://dx.doi.org/10.1097/ICU.0000000000000359
https://repositorio.unifesp.br/handle/11600/54688
Resumo: Purpose 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.
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spelling CRISPR applications in ophthalmologic PINION genome surgeryadeno-associated virusCRISPR-Casembryonic stem cellsinduced pluripotent stem cellsprecision medicinePurpose 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.Columbia Univ, Dept Ophthalmol, Med Ctr, Jonas Childrens Vis Care, 635 West 165th St,5th Floor, New York, NY 10032 USAColumbia Univ, Dept Ophthalmol, Med Ctr, Bernard & Shirlee Brown Glaucoma Lab, 635 West 165th St,5th Floor, New York, NY 10032 USANew York Presbyterian Hosp, Edward S Harkness Eye Inst, 635 West 165th St,5th Floor, New York, NY 10032 USAUniv Fed Espirito Santo, Dept Ophthalmol, Vitoria, BrazilUniv Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, BrazilSuny Downstate Med Ctr, Brooklyn, NY 11203 USAShanghai Jiao Tong Univ, Dept Ophthalmol, Xin Hua Hosp, Shanghai, Peoples R ChinaColumbia Univ, Inst Human Nutr, Dept Pathol & Cell Biol, Coll Phys & Surg, New York, NY 10032 USAUniv Fed Sao Paulo, Dept Ophthalmol, Sao Paulo, BrazilWeb of ScienceNational Institutes of HealthNational Cancer Institute CoreResearch to Prevent Blindness (RPB) Physician-Scientist AwardRPB, New York, NY, USAICO-Retina Research Foundation Helmerich FellowshipsRetina Research FoundationICO Foundation (International Council of Ophthalmology)RPB medical student fellowshipTistou and Charlotte Kerstan FoundationSchneeweiss Stem Cell Fund, New York StateFoundation Fighting Blindness New York Regional Research Center GrantNational Institutes of Health: 5P30EY019007National Institutes of Health: R01EY018213National Institutes of Health: R01EY024698National Institutes of Health: 1R01EY026682National Institutes of Health: R21AG050437National Cancer Institute Core: 5P30CA013696Schneeweiss Stem Cell Fund, New York State: C029572Foundation Fighting Blindness New York Regional Research Center Grant: C-NY05-0705-0312Lippincott Williams & Wilkins2020-07-17T14:02:13Z2020-07-17T14:02:13Z2017info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersion252-259http://dx.doi.org/10.1097/ICU.0000000000000359Current Opinion In Ophthalmology. Philadelphia, v. 28, n. 3, p. 252-259, 2017.10.1097/ICU.00000000000003591040-8738https://repositorio.unifesp.br/handle/11600/54688WOS:000399390800008ark:/48912/001300000z5s3engCurrent Opinion In OphthalmologyPhiladelphiainfo:eu-repo/semantics/openAccessCabral, Thiago [UNIFESP]DiCarlo, James E.Justus, SallySengillo, Jesse D.Xu, YuTsang, Stephen H.reponame:Repositório Institucional da UNIFESPinstname:Universidade Federal de São Paulo (UNIFESP)instacron:UNIFESP2022-02-07T21:55:48Zoai:repositorio.unifesp.br/:11600/54688Repositório InstitucionalPUBhttp://www.repositorio.unifesp.br/oai/requestbiblioteca.csp@unifesp.bropendoar:34652024-12-11T20:44:42.194854Repositório Institucional da UNIFESP - Universidade Federal de São Paulo (UNIFESP)false
dc.title.none.fl_str_mv CRISPR applications in ophthalmologic PINION genome surgery
title CRISPR applications in ophthalmologic PINION genome surgery
spellingShingle CRISPR applications in ophthalmologic PINION genome surgery
Cabral, Thiago [UNIFESP]
adeno-associated virus
CRISPR-Cas
embryonic stem cells
induced pluripotent stem cells
precision medicine
title_short CRISPR applications in ophthalmologic PINION genome surgery
title_full CRISPR applications in ophthalmologic PINION genome surgery
title_fullStr CRISPR applications in ophthalmologic PINION genome surgery
title_full_unstemmed CRISPR applications in ophthalmologic PINION genome surgery
title_sort CRISPR applications in ophthalmologic PINION genome surgery
author Cabral, Thiago [UNIFESP]
author_facet Cabral, Thiago [UNIFESP]
DiCarlo, James E.
Justus, Sally
Sengillo, Jesse D.
Xu, Yu
Tsang, Stephen H.
author_role author
author2 DiCarlo, James E.
Justus, Sally
Sengillo, Jesse D.
Xu, Yu
Tsang, Stephen H.
author2_role author
author
author
author
author
dc.contributor.author.fl_str_mv Cabral, Thiago [UNIFESP]
DiCarlo, James E.
Justus, Sally
Sengillo, Jesse D.
Xu, Yu
Tsang, Stephen H.
dc.subject.por.fl_str_mv adeno-associated virus
CRISPR-Cas
embryonic stem cells
induced pluripotent stem cells
precision medicine
topic adeno-associated virus
CRISPR-Cas
embryonic stem cells
induced pluripotent stem cells
precision medicine
description Purpose 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.
publishDate 2017
dc.date.none.fl_str_mv 2017
2020-07-17T14:02:13Z
2020-07-17T14:02:13Z
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://dx.doi.org/10.1097/ICU.0000000000000359
Current Opinion In Ophthalmology. Philadelphia, v. 28, n. 3, p. 252-259, 2017.
10.1097/ICU.0000000000000359
1040-8738
https://repositorio.unifesp.br/handle/11600/54688
WOS:000399390800008
dc.identifier.dark.fl_str_mv ark:/48912/001300000z5s3
url http://dx.doi.org/10.1097/ICU.0000000000000359
https://repositorio.unifesp.br/handle/11600/54688
identifier_str_mv Current Opinion In Ophthalmology. Philadelphia, v. 28, n. 3, p. 252-259, 2017.
10.1097/ICU.0000000000000359
1040-8738
WOS:000399390800008
ark:/48912/001300000z5s3
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Current Opinion In Ophthalmology
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv 252-259
dc.coverage.none.fl_str_mv Philadelphia
dc.publisher.none.fl_str_mv Lippincott Williams & Wilkins
publisher.none.fl_str_mv Lippincott Williams & Wilkins
dc.source.none.fl_str_mv reponame:Repositório Institucional da UNIFESP
instname:Universidade Federal de São Paulo (UNIFESP)
instacron:UNIFESP
instname_str Universidade Federal de São Paulo (UNIFESP)
instacron_str UNIFESP
institution UNIFESP
reponame_str Repositório Institucional da UNIFESP
collection Repositório Institucional da UNIFESP
repository.name.fl_str_mv Repositório Institucional da UNIFESP - Universidade Federal de São Paulo (UNIFESP)
repository.mail.fl_str_mv biblioteca.csp@unifesp.br
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