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CMT1A current gene therapy approaches and promising biomarkers

18 Nov 2022

Abstract excerpt

Charcot-Marie-Tooth neuropathies (CMT) constitute a group of common but highly heterogeneous, non-syndromic genetic disorders affecting predominantly the peripheral nervous system. CMT type 1A (CMT1A) is the most frequent type and accounts for almost ~50% of all diagnosed CMT cases. CMT1A results from the duplication of the peripheral myelin protein 22 (PMP22) gene. Overexpression of PMP22 protein overloads the...

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PMID: 36571339
DOI: 10.4103/1673-5374.361538

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