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Treatment of a Mouse Model of ALS by In Vivo Base Editing

13 Jan 2020

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a debilitating and fatal disorder that can be caused by mutations in the superoxide dismutase 1 (SOD1) gene. Although ALS is currently incurable, CRISPR base editors hold the potential to treat the disease through their ability to create nonsense mutations that can permanently disable the expression of the mutant SOD1 gene. However, the restrictive carrying capacity of...

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PMID: 31991108
DOI: 10.1016/j.ymthe.2020.01.005

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