Commentary 10.1172/JCI98617

Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A

Michael E. Shy

Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA.

Address correspondence to: Michael E. Shy, 200 Hawkins Drive, Carver College of Medicine, Department of Neurology, University of Iowa, Iowa City, Iowa 52242, USA. Phone: 319.353.5097; Email: michael-shy@uiowa.edu.

Find articles by Shy, M. in: JCI | PubMed | Google Scholar

First published December 4, 2017 - More info

Published in Volume 128, Issue 1 (January 2, 2018)
J Clin Invest. 2018;128(1):110–112. https://doi.org/10.1172/JCI98617.
Copyright © 2018, American Society for Clinical Investigation

First published December 4, 2017

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.

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