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Fig. 1. Types of gene therapy and therapeutic strategies for inherited peripheral neuropathy. Based on the mode of inheritance IPN treatment can be categorized into three groups. For recessively inherited cases, gene replacement, the delivery of a functional gene, was applied to compensate for the lack of functional protein. For dominantly inherited IPN, NT-3, a neuroprotective gene, was used to ameliorate the disease phenotype. The feasibility of mutant allele-specific was also validated for suppressing the gain-of-toxicity mutant protein in dominantly inherited IPN. For CMT1A caused by the duplication of PMP22, various approaches have been developed to normalize the expression level of PMP22. Antisense oligonucleotide (ASO) or miRNA targeting the 3'-untranslated region (3'-UTR) of PMP22, and disruption of the TATA-box could effectively reduce the protein level in vivo.
Exp Neurobiol 2020;29:177~188
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