PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models

Abstract excerpt

Charcot-Marie-Tooth disease type 1A (CMT1A) is caused by duplication of peripheral myelin protein 22 (PMP22) and is the most common hereditary peripheral neuropathy. CMT1A is characterized by demyelination and axonal loss, which underlie slowed motor nerve conduction velocity (MNCV) and reduced compound muscle action potentials (CMAP) in patients. There is currently no known treatment for this disease. Here, we...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Botulinum Toxin and Related Neurological Disorders
• Share to Genetic Neurodegenerative Diseases
• Share to Hereditary Neurological Disorders