https://www.jci.org/articles/view/98617
https://www.ncbi.nlm.nih.gov/pubmed/29199996
Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A.
J Clin Invest. 2018 Jan 2;128(1):110-112. doi: 10.1172/JCI98617. Epub 2017 Dec 4.
Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A.
Shy ME.
Abstract
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 therapeutic approaches for CMT1A and lay the groundwork for clinical trials in humans afflicted with this chronic, debilitating neurodegenerative disease.
PMID: 29199996 PMCID: PMC5749496 DOI: 10.1172/JCI98617
Free PMC Article
