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Usage Information

A hit for base editing: treatment of developmental epilepsy in a mouse model
Sophie F. Hill, Ethan M. Goldberg
Sophie F. Hill, Ethan M. Goldberg
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Commentary

A hit for base editing: treatment of developmental epilepsy in a mouse model

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Abstract

CRISPR/Cas9 base editing holds the potential to treat disease caused by single-nucleotide variants. In contrast with conventional CRISPR/Cas9 approaches, base editing enzymatically induces precise DNA alterations and can directly correct disease-causing variants. In this issue of JCI, Reever et al. used base editing to treat a mouse model of a severe neurodevelopmental disorder caused by a pathogenic missense variant in the voltage-gated sodium channel gene SCN8A. This work represents a starting point for the further refinement of base editing to treat genetic epilepsy.

Authors

Sophie F. Hill, Ethan M. Goldberg

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ISSN: 0021-9738 (print), 1558-8238 (online)

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