Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS

Deng et al. assess the effects of CRISPR/Cas9-mediated genome editing in two transgenic mouse models of amyotrophic lateral sclerosis (ALS) for up to 2 years. They find that the genomic editing prevented the development of ALS-like pathology without any notable side-effects, which provides preclinic...

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Autores principales:	Han-Xiang Deng, Hong Zhai, Yong Shi, Guoxiang Liu, Jessica Lowry, Bin Liu, Éanna B. Ryan, Jianhua Yan, Yi Yang, Nigel Zhang, Zhihua Yang, Erdong Liu, Yongchao C. Ma, Teepu Siddique
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Biology (General) QH301-705.5
Acceso en línea:	https://doaj.org/article/e548fc08a9654fe99fd22baa46408209
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spelling	oai:doaj.org-article:e548fc08a9654fe99fd22baa464082092021-12-02T11:45:00ZEfficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS10.1038/s42003-021-01942-42399-3642https://doaj.org/article/e548fc08a9654fe99fd22baa464082092021-03-01T00:00:00Zhttps://doi.org/10.1038/s42003-021-01942-4https://doaj.org/toc/2399-3642Deng et al. assess the effects of CRISPR/Cas9-mediated genome editing in two transgenic mouse models of amyotrophic lateral sclerosis (ALS) for up to 2 years. They find that the genomic editing prevented the development of ALS-like pathology without any notable side-effects, which provides preclinical evidence of the effectiveness and long-term safety of the CRISPR/Cas9 therapeutic approach.Han-Xiang DengHong ZhaiYong ShiGuoxiang LiuJessica LowryBin LiuÉanna B. RyanJianhua YanYi YangNigel ZhangZhihua YangErdong LiuYongchao C. MaTeepu SiddiqueNature PortfolioarticleBiology (General)QH301-705.5ENCommunications Biology, Vol 4, Iss 1, Pp 1-11 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Biology (General) QH301-705.5
spellingShingle	Biology (General) QH301-705.5 Han-Xiang Deng Hong Zhai Yong Shi Guoxiang Liu Jessica Lowry Bin Liu Éanna B. Ryan Jianhua Yan Yi Yang Nigel Zhang Zhihua Yang Erdong Liu Yongchao C. Ma Teepu Siddique Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
description	Deng et al. assess the effects of CRISPR/Cas9-mediated genome editing in two transgenic mouse models of amyotrophic lateral sclerosis (ALS) for up to 2 years. They find that the genomic editing prevented the development of ALS-like pathology without any notable side-effects, which provides preclinical evidence of the effectiveness and long-term safety of the CRISPR/Cas9 therapeutic approach.
format	article
author	Han-Xiang Deng Hong Zhai Yong Shi Guoxiang Liu Jessica Lowry Bin Liu Éanna B. Ryan Jianhua Yan Yi Yang Nigel Zhang Zhihua Yang Erdong Liu Yongchao C. Ma Teepu Siddique
author_facet	Han-Xiang Deng Hong Zhai Yong Shi Guoxiang Liu Jessica Lowry Bin Liu Éanna B. Ryan Jianhua Yan Yi Yang Nigel Zhang Zhihua Yang Erdong Liu Yongchao C. Ma Teepu Siddique
author_sort	Han-Xiang Deng
title	Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
title_short	Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
title_full	Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
title_fullStr	Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
title_full_unstemmed	Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
title_sort	efficacy and long-term safety of crispr/cas9 genome editing in the sod1-linked mouse models of als
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/e548fc08a9654fe99fd22baa46408209
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Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS

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