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

25 Mar 2021

Abstract excerpt

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 sclerosis (ALS) transgenic mouse models prevented the development of ALS-like disease and pathology. The disease-linked...

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PMID: 33767386
DOI: 10.1038/s42003-021-01942-4

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