Cas9 immunity creates challenges for CRISPR gene editing therapies

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology1,2 that utilizes archaeal and bacterial Cas9 nucleases to introduce double-stranded breaks in DNA at targeted sites. These breaks can be used to remove, replace, or add pieces of DNA. Whil...

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Autores principales:	Julie M. Crudele, Jeffrey S. Chamberlain
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2018
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/e6e102c06f9f4413a3b861226fafa27a
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id	oai:doaj.org-article:e6e102c06f9f4413a3b861226fafa27a
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spelling	oai:doaj.org-article:e6e102c06f9f4413a3b861226fafa27a2021-12-02T17:33:03ZCas9 immunity creates challenges for CRISPR gene editing therapies10.1038/s41467-018-05843-92041-1723https://doaj.org/article/e6e102c06f9f4413a3b861226fafa27a2018-08-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-05843-9https://doaj.org/toc/2041-1723Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology1,2 that utilizes archaeal and bacterial Cas9 nucleases to introduce double-stranded breaks in DNA at targeted sites. These breaks can be used to remove, replace, or add pieces of DNA. While not the first genome editor, CRISPR-Cas9 is efficient and cost-effective because cutting is guided by a strand of RNA rather than a protein. The potential uses in health care are plentiful, from disrupting dominant genes that cause cancer3 to repairing mutated genes that cause genetic diseases, such as muscular dystrophy4. Therapeutic approaches based on this technology fill the preclinical pipeline, and rely on the use of viral vectors to deliver the Cas9 gene and guide RNA to a gene of interest. However, concerns regarding the safety and efficacy of CRISPR-Cas9 use in gene therapy remain. A pre-print released prior to peer review has recently underlined the question of whether immunological responses to Cas9 may negatively impact its clinical use5. Here we discuss the implications of this finding for the application of CRISPR/Cas in gene therapy.Julie M. CrudeleJeffrey S. ChamberlainNature PortfolioarticleScienceQENNature Communications, Vol 9, Iss 1, Pp 1-3 (2018)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Julie M. Crudele Jeffrey S. Chamberlain Cas9 immunity creates challenges for CRISPR gene editing therapies
description	Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology1,2 that utilizes archaeal and bacterial Cas9 nucleases to introduce double-stranded breaks in DNA at targeted sites. These breaks can be used to remove, replace, or add pieces of DNA. While not the first genome editor, CRISPR-Cas9 is efficient and cost-effective because cutting is guided by a strand of RNA rather than a protein. The potential uses in health care are plentiful, from disrupting dominant genes that cause cancer3 to repairing mutated genes that cause genetic diseases, such as muscular dystrophy4. Therapeutic approaches based on this technology fill the preclinical pipeline, and rely on the use of viral vectors to deliver the Cas9 gene and guide RNA to a gene of interest. However, concerns regarding the safety and efficacy of CRISPR-Cas9 use in gene therapy remain. A pre-print released prior to peer review has recently underlined the question of whether immunological responses to Cas9 may negatively impact its clinical use5. Here we discuss the implications of this finding for the application of CRISPR/Cas in gene therapy.
format	article
author	Julie M. Crudele Jeffrey S. Chamberlain
author_facet	Julie M. Crudele Jeffrey S. Chamberlain
author_sort	Julie M. Crudele
title	Cas9 immunity creates challenges for CRISPR gene editing therapies
title_short	Cas9 immunity creates challenges for CRISPR gene editing therapies
title_full	Cas9 immunity creates challenges for CRISPR gene editing therapies
title_fullStr	Cas9 immunity creates challenges for CRISPR gene editing therapies
title_full_unstemmed	Cas9 immunity creates challenges for CRISPR gene editing therapies
title_sort	cas9 immunity creates challenges for crispr gene editing therapies
publisher	Nature Portfolio
publishDate	2018
url	https://doaj.org/article/e6e102c06f9f4413a3b861226fafa27a
work_keys_str_mv	AT juliemcrudele cas9immunitycreateschallengesforcrisprgeneeditingtherapies AT jeffreyschamberlain cas9immunitycreateschallengesforcrisprgeneeditingtherapies
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Cas9 immunity creates challenges for CRISPR gene editing therapies

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