Journal article
Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A
The Journal of clinical investigation, Vol.128(1), pp.110-112
01/02/2018
DOI: 10.1172/JCI98617
PMCID: PMC5749496
PMID: 29199996
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.
Details
- Title: Subtitle
- Antisense oligonucleotides offer hope to patients with Charcot-Marie-Tooth disease type 1A
- Creators
- Michael E Shy - Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA
- Resource Type
- Journal article
- Publication Details
- The Journal of clinical investigation, Vol.128(1), pp.110-112
- Publisher
- American Society for Clinical Investigation
- DOI
- 10.1172/JCI98617
- PMID
- 29199996
- PMCID
- PMC5749496
- ISSN
- 0021-9738
- eISSN
- 1558-8238
- Language
- English
- Date published
- 01/02/2018
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984070654302771
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