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Peripheral neuropathies

Antisense therapy for Charcot–Marie–Tooth disease?

Nature Reviews Neurology volume 14, page 64 (2018)Cite this article

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CMT encompasses a group of inherited peripheral neuropathies that are characterized by progressive muscle weakness and numbness of sensation. No effective therapies are currently available for these conditions. The most common form of CMT — CMT1A — is caused by a duplication of PMP22, which encodes peripheral myelin protein 22, a major component of myelin.

In rodent models of CMT1A, overexpression of PMP22 is sufficient to cause demyelination. Subsequent reduction of this overexpression results in remyelination, suggesting that lowering the expression levels of PMP22 is a viable therapeutic strategy in patients with CMT1A.

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References

  1. Zhao, H. T. et al. PMP22 antisense oligonucleotides reverse Charcot–Marie–Tooth disease type 1A features in rodent models. J. Clin. Invest. http://dx.doi.org/10.1172/JCI96499 (2017)

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Authors
  1. Charlotte Ridler
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Ridler, C. Antisense therapy for Charcot–Marie–Tooth disease?. Nat Rev Neurol 14, 64 (2018). https://doi.org/10.1038/nrneurol.2017.182

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