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

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...

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Bibliografiske detaljer
Udgivet i:J Clin Invest
Hovedforfatter: Svaren, John
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2021
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8159695/
https://ncbi.nlm.nih.gov/pubmed/34060476
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI149492
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