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

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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