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