Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
oleh: 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
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2021-03-01 |
Deskripsi
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.