Effects of gene replacement therapy in a murine model of Charcot-Marie-Tooth 4C neuropathy

Takeaway

  • SH3TC2 gene replacement improved motor performance, motor nerve conduction velocities, myelination, and disease associated morphological abnormalities in sciatic nerves and lumbar roots in a murine model of Charcot-Marie-Tooth 4C neuropathy.

Why this matters

  • Proof of principle that gene therapy targeted to Schwann cells alleviate changes induced by Charcot-Marie-Tooth disease in animal models and has the potential to be effective not only in this condition but in other demyelinating inherited neuropathies.