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Engineered Viruses as Genome Editing Devices

Genome editing based on sequence-specific designer nucleases, also known as programmable nucleases, seeks to modify in a targeted and precise manner the genetic information content of living cells. Delivering into cells designer nucleases alone or together with donor DNA templates, which serve as su...

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Autores principales: Chen, Xiaoyu, Gonçalves, Manuel A F V
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786910/
https://www.ncbi.nlm.nih.gov/pubmed/26336974
http://dx.doi.org/10.1038/mt.2015.164
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author Chen, Xiaoyu
Gonçalves, Manuel A F V
author_facet Chen, Xiaoyu
Gonçalves, Manuel A F V
author_sort Chen, Xiaoyu
collection PubMed
description Genome editing based on sequence-specific designer nucleases, also known as programmable nucleases, seeks to modify in a targeted and precise manner the genetic information content of living cells. Delivering into cells designer nucleases alone or together with donor DNA templates, which serve as surrogate homologous recombination (HR) substrates, can result in gene knockouts or gene knock-ins, respectively. As engineered replication-defective viruses, viral vectors are having an increasingly important role as delivery vehicles for donor DNA templates and designer nucleases, namely, zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated Cas9 (CRISPR−Cas9) nucleases, also known as RNA-guided nucleases (RGNs). We review this dual role played by engineered viral particles on genome editing while focusing on their main scaffolds, consisting of lentiviruses, adeno-associated viruses, and adenoviruses. In addition, the coverage of the growing body of research on the repurposing of viral vectors as delivery systems for genome editing tools is complemented with information regarding their main characteristics, pros, and cons. Finally, this information is framed by a concise description of the chief principles, tools, and applications of the genome editing field as a whole.
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spelling pubmed-47869102016-03-16 Engineered Viruses as Genome Editing Devices Chen, Xiaoyu Gonçalves, Manuel A F V Mol Ther Review Genome editing based on sequence-specific designer nucleases, also known as programmable nucleases, seeks to modify in a targeted and precise manner the genetic information content of living cells. Delivering into cells designer nucleases alone or together with donor DNA templates, which serve as surrogate homologous recombination (HR) substrates, can result in gene knockouts or gene knock-ins, respectively. As engineered replication-defective viruses, viral vectors are having an increasingly important role as delivery vehicles for donor DNA templates and designer nucleases, namely, zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated Cas9 (CRISPR−Cas9) nucleases, also known as RNA-guided nucleases (RGNs). We review this dual role played by engineered viral particles on genome editing while focusing on their main scaffolds, consisting of lentiviruses, adeno-associated viruses, and adenoviruses. In addition, the coverage of the growing body of research on the repurposing of viral vectors as delivery systems for genome editing tools is complemented with information regarding their main characteristics, pros, and cons. Finally, this information is framed by a concise description of the chief principles, tools, and applications of the genome editing field as a whole. Nature Publishing Group 2016-03 2015-10-06 /pmc/articles/PMC4786910/ /pubmed/26336974 http://dx.doi.org/10.1038/mt.2015.164 Text en Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy http://creativecommons.org/licenses/by-nc-sa/4.0/ This work is licensed under a Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International License. The images or other third party material in this article are included in the article's Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-sa/4.0/
spellingShingle Review
Chen, Xiaoyu
Gonçalves, Manuel A F V
Engineered Viruses as Genome Editing Devices
title Engineered Viruses as Genome Editing Devices
title_full Engineered Viruses as Genome Editing Devices
title_fullStr Engineered Viruses as Genome Editing Devices
title_full_unstemmed Engineered Viruses as Genome Editing Devices
title_short Engineered Viruses as Genome Editing Devices
title_sort engineered viruses as genome editing devices
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786910/
https://www.ncbi.nlm.nih.gov/pubmed/26336974
http://dx.doi.org/10.1038/mt.2015.164
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