CRISPR applications in ophthalmologic PINION genome surgery
Autor(a) principal: | |
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Data de Publicação: | 2017 |
Outros Autores: | , , , , |
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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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 |
_version_ |
1818602538086170624 |