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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.


Original article

  1. Schiza, N. et al. Gene replacement therapy in a model of Charcot–Marie–Tooth 4C neuropathy. Brain (2019)

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Correspondence to Ian Fyfe.

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Fyfe, I. Gene therapy is effective for CMT in mice. Nat Rev Neurol 15, 308 (2019).

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