Ontology highlight
ABSTRACT:
SUBMITTER: Shy ME
PROVIDER: S-EPMC5749496 | biostudies-literature | 2018 Jan
REPOSITORIES: biostudies-literature
The Journal of clinical investigation 20171204 1
Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common heritable peripheral neuropathy and results from a duplication on chromosome 17 that results in an extra copy and increased dosage of peripheral myelin protein 22 (PMP22). Zhao et al., in this issue of the JCI, successfully utilized antisense oligonucleotides (ASOs) to reduce PMP22 and ameliorated neuropathy in both mouse and rat models of CMT1A. These data confirm that strategies to reduce PMP22 have potential as effective therapeut ...[more]