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Article

Adeno-associated virus gene therapy to the rescue for Charcot-Marie-Tooth disease type 4J.

The Journal of clinical investigation - 1 Jun 2021

Svaren John

Abstract excerpt

The genetic peripheral neuropathy known as Charcot-Marie-Tooth disease type 4J (CMT4J) is caused by recessive mutations in the FIG4 gene. The transformational success of adeno-associated virus (AAV) gene therapy for spinal muscular atrophy has generated substantial interest in using this approach to create similar treatments for CMT. In this issue of the JCI, Presa et al. provide a preclinical demonstration of...

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PMID: 34060476
DOI: 10.1172/JCI149492
PMCID: PMC8159695

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