Charcot-Marie-Tooth neuropathies: Current gene therapy advances and the route toward translation.

Abstract excerpt

Charcot-Marie-Tooth (CMT) neuropathies are a group of genetically and phenotypically heterogeneous disorders that predominantly affect the peripheral nervous system. Unraveling the genetic and molecular mechanisms, as well as the cellular effects of CMT mutations, has facilitated the development of promising gene therapy approaches. Proposed gene therapy treatments for CMTs include virally or non-virally mediated...

Topics

• Animals
• Charcot-Marie-Tooth Disease
• Mutation
• Phenotype
• Schwann Cells