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Gene Editing and Genotoxicity: Targeting the Off-Targets

Gene editing technologies show great promise for application to human disease as a result of rapid developments in targeting tools notably based on ZFN, TALEN, and CRISPR-Cas systems. Precise modification of a DNA sequence is now possible in mature human somatic cells including stem and progenitor c...

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Autores principales: Blattner, Georges, Cavazza, Alessia, Thrasher, Adrian J., Turchiano, Giandomenico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525370/
https://www.ncbi.nlm.nih.gov/pubmed/34713236
http://dx.doi.org/10.3389/fgeed.2020.613252
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author Blattner, Georges
Cavazza, Alessia
Thrasher, Adrian J.
Turchiano, Giandomenico
author_facet Blattner, Georges
Cavazza, Alessia
Thrasher, Adrian J.
Turchiano, Giandomenico
author_sort Blattner, Georges
collection PubMed
description Gene editing technologies show great promise for application to human disease as a result of rapid developments in targeting tools notably based on ZFN, TALEN, and CRISPR-Cas systems. Precise modification of a DNA sequence is now possible in mature human somatic cells including stem and progenitor cells with increasing degrees of efficiency. At the same time new technologies are required to evaluate their safety and genotoxicity before widespread clinical application can be confidently implemented. A number of methodologies have now been developed in an attempt to predict expected and unexpected modifications occurring during gene editing. This review surveys the techniques currently available as state of the art, highlighting benefits and limitations, and discusses approaches that may achieve sufficient accuracy and predictability for application in clinical settings.
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spelling pubmed-85253702021-10-27 Gene Editing and Genotoxicity: Targeting the Off-Targets Blattner, Georges Cavazza, Alessia Thrasher, Adrian J. Turchiano, Giandomenico Front Genome Ed Genome Editing Gene editing technologies show great promise for application to human disease as a result of rapid developments in targeting tools notably based on ZFN, TALEN, and CRISPR-Cas systems. Precise modification of a DNA sequence is now possible in mature human somatic cells including stem and progenitor cells with increasing degrees of efficiency. At the same time new technologies are required to evaluate their safety and genotoxicity before widespread clinical application can be confidently implemented. A number of methodologies have now been developed in an attempt to predict expected and unexpected modifications occurring during gene editing. This review surveys the techniques currently available as state of the art, highlighting benefits and limitations, and discusses approaches that may achieve sufficient accuracy and predictability for application in clinical settings. Frontiers Media S.A. 2020-12-10 /pmc/articles/PMC8525370/ /pubmed/34713236 http://dx.doi.org/10.3389/fgeed.2020.613252 Text en Copyright © 2020 Blattner, Cavazza, Thrasher and Turchiano. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Genome Editing
Blattner, Georges
Cavazza, Alessia
Thrasher, Adrian J.
Turchiano, Giandomenico
Gene Editing and Genotoxicity: Targeting the Off-Targets
title Gene Editing and Genotoxicity: Targeting the Off-Targets
title_full Gene Editing and Genotoxicity: Targeting the Off-Targets
title_fullStr Gene Editing and Genotoxicity: Targeting the Off-Targets
title_full_unstemmed Gene Editing and Genotoxicity: Targeting the Off-Targets
title_short Gene Editing and Genotoxicity: Targeting the Off-Targets
title_sort gene editing and genotoxicity: targeting the off-targets
topic Genome Editing
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525370/
https://www.ncbi.nlm.nih.gov/pubmed/34713236
http://dx.doi.org/10.3389/fgeed.2020.613252
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