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

CRISPR/Cas9-mediated genome editing provides potential for therapeutic development. Efficacy and long-term safety represent major concerns that remain to be adequately addressed in preclinical studies. Here we show that CRISPR/Cas9-mediated genome editing in two distinct SOD1-amyotrophic lateral scl...

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Udgivet i:Commun Biol
Main Authors: Deng, Han-Xiang, Zhai, Hong, Shi, Yong, Liu, Guoxiang, Lowry, Jessica, Liu, Bin, Ryan, Éanna B., Yan, Jianhua, Yang, Yi, Zhang, Nigel, Yang, Zhihua, Liu, Erdong, Ma, Yongchao C., Siddique, Teepu
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7994668/
https://ncbi.nlm.nih.gov/pubmed/33767386
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s42003-021-01942-4
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