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Charcot-Marie-Tooth disease type 1A (CMT1A), caused by duplication of the peripheral myelin protein 22 ( PMP22 ) gene, and CMT1B, caused by mutations in myelin protein zero ( MPZ ) gene, are the two most common forms of demyelinating CMT (CMT1), and no treatments are available for either. Prior studies of the Mpz Ser63del mouse model of CMT1B have demonstrated that protein misfolding, endoplasmic reticulum (ER) retention and a...
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