Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

PERIPHERAL NEUROPATHIES

Gene therapy is effective for CMT in mice

Virus-mediated gene therapy has been used to treat a mouse model of Charcot–Marie–Tooth disease (CMT) type 4C in a recently published proof-of-principle study. Natasa Schiza and colleagues developed a lentiviral vector that was administered by intrathecal injection to mice in which the Sh3tc2 gene — mutation or truncation of which causes CMT type 4C — was knocked out. At 8 weeks after the injection, motor performance was better in mice that had been treated with the gene therapy than in those that had been treated with a mock lentivirus. Nerve conduction velocity, myelin morphology and nodal molecular architecture were also all improved, and blood levels of neurofilament light — a marker of axonal degeneration — were reduced. The study indicates that viral gene replacement therapy that targets Schwann cells could be used to treat CMT type 4C and possibly other similar demyelinating neuropathies.

References

Original article

  1. Schiza, N. et al. Gene replacement therapy in a model of Charcot–Marie–Tooth 4C neuropathy. Brain https://doi.org/10.1093/brain/awz064 (2019)

    Article  PubMed  PubMed Central  Google Scholar 

Download references

Author information

Affiliations

Authors

Corresponding author

Correspondence to Ian Fyfe.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Fyfe, I. Gene therapy is effective for CMT in mice. Nat Rev Neurol 15, 308 (2019). https://doi.org/10.1038/s41582-019-0193-1

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41582-019-0193-1

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing