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Article

Gene therapy and other novel treatment approaches for Charcot-Marie-Tooth disease.

Neuromuscular disorders : NMD - 1 Aug 2023

Pisciotta Chiara, Pareyson Davide

Abstract excerpt

There is still no effective drug treatment available for Charcot-Marie-Tooth disease (CMT). Current management relies on rehabilitation therapy, surgery for skeletal deformities, and symptomatic treatment. The challenge is to find disease-modifying therapies. Several approaches, including gene silencing (by means of ASO, siRNA, shRNA, miRNA, CRISPR-Cas9 editing), to counteract the PMP22 gene overexpression in the...

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PMID: 37455204
DOI: 10.1016/j.nmd.2023.07.001

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