Cell-targeted Gene Therapy May Reverse Nerve Damage in CMTX1

Cell-targeted Gene Therapy May Reverse Nerve Damage in CMTX1

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Cell-targeted Gene Therapy May Reverse Nerve Damage in CMTX1

A single dose of a gene therapy delivering a working copy of GJB1 — the gene mutated in X-linked Charcot-Marie-Tooth type 1 disease (CMTX1) — to myelin-producing cells prevented or even reversed myelin loss and motor function impairment in a mouse model of CMTX1. Notably, these benefits were observed not only when the therapy was given before symptom onset, but also at later stages of the disease. According to researchers, these results highlight the therapeutic…

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