Investigational CMT1A Gene Therapy Eased Myelin Loss in Mice

Investigational CMT1A Gene Therapy Eased Myelin Loss in Mice

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Investigational CMT1A Gene Therapy Eased Myelin Loss in Mice

A potential gene therapy for Charcot-Marie-Tooth disease type 1A (CMT1A) has shown promising results in a two-year study using a mouse model of the disease. According to a press release from the CMT Research Foundation, which is funding the study, the gene therapy led to marked reductions in the levels of PMP22, the protein involved in CMT1A, in peripheral nerves compared to untreated mice, used as controls. Peripheral nerves send sensory and motor information from the brain…

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