Article
Gene therapy approaches targeting Schwann cells for demyelinating neuropathies.
Brain research - 1 Feb 2020
Sargiannidou Irene, Kagiava Alexia, Kleopa Kleopas A
Abstract excerpt
Charcot-Marie-Tooth disease (CMT) encompasses numerous genetically heterogeneous inherited neuropathies, which together are one of the commonest neurogenetic disorders. Axonal CMT types result from mutations in neuronally expressed genes, whereas demyelinating CMT forms mostly result from mutations in genes expressed by myelinating Schwann cells. The demyelinating forms are the most common, and may be caused by...
Topics
- Charcot-Marie-Tooth Disease
- Connexins
- Demyelinating Diseases
- Genetic Therapy
- Humans
- Intracellular Signaling Peptides and Proteins
- Mutation
- Promoter Regions, Genetic
- Schwann Cells
- Gap Junction beta-1 Protein