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Genome-editing Technologies for Gene and Cell Therapy
Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the corre...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786923/ https://www.ncbi.nlm.nih.gov/pubmed/26755333 http://dx.doi.org/10.1038/mt.2016.10 |
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author | Maeder, Morgan L Gersbach, Charles A |
author_facet | Maeder, Morgan L Gersbach, Charles A |
author_sort | Maeder, Morgan L |
collection | PubMed |
description | Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the genome, and the removal of deleterious genes or genome sequences. This review presents the mechanisms of different genome-editing strategies and describes each of the common nuclease-based platforms, including zinc finger nucleases, transcription activator-like effector nucleases (TALENs), meganucleases, and the CRISPR/Cas9 system. We then summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. The current challenges and future prospects for genome editing as a transformative technology for gene and cell therapy are also discussed. |
format | Online Article Text |
id | pubmed-4786923 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2016 |
publisher | Nature Publishing Group |
record_format | MEDLINE/PubMed |
spelling | pubmed-47869232016-03-16 Genome-editing Technologies for Gene and Cell Therapy Maeder, Morgan L Gersbach, Charles A Mol Ther Review Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the genome, and the removal of deleterious genes or genome sequences. This review presents the mechanisms of different genome-editing strategies and describes each of the common nuclease-based platforms, including zinc finger nucleases, transcription activator-like effector nucleases (TALENs), meganucleases, and the CRISPR/Cas9 system. We then summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. The current challenges and future prospects for genome editing as a transformative technology for gene and cell therapy are also discussed. Nature Publishing Group 2016-03 2016-02-16 /pmc/articles/PMC4786923/ /pubmed/26755333 http://dx.doi.org/10.1038/mt.2016.10 Text en Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy http://creativecommons.org/licenses/by/4.0/ This work is licensed under a Creative Commons Attribution 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/4.0/ |
spellingShingle | Review Maeder, Morgan L Gersbach, Charles A Genome-editing Technologies for Gene and Cell Therapy |
title | Genome-editing Technologies for Gene and Cell Therapy |
title_full | Genome-editing Technologies for Gene and Cell Therapy |
title_fullStr | Genome-editing Technologies for Gene and Cell Therapy |
title_full_unstemmed | Genome-editing Technologies for Gene and Cell Therapy |
title_short | Genome-editing Technologies for Gene and Cell Therapy |
title_sort | genome-editing technologies for gene and cell therapy |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786923/ https://www.ncbi.nlm.nih.gov/pubmed/26755333 http://dx.doi.org/10.1038/mt.2016.10 |
work_keys_str_mv | AT maedermorganl genomeeditingtechnologiesforgeneandcelltherapy AT gersbachcharlesa genomeeditingtechnologiesforgeneandcelltherapy |