Intravenous Administration of an AAV9 Vector Ubiquitously Expressing C1ORF194 Gene Improved CMT-like Neuropathy in C1ORF194-/- Mice

Medicine, Basic Medical Sciences, Neurology, Life Sciences, Cell Biology, Biochemistry, Immunology, Genetics,
CMT disease   C1ORF194   gene therapy   AAV9   neuropathy   motor ability   sensory nerves  

Research Report on the Improvement of CMT-like Neuropathy via AAV9 Vector-based C1ORF194 Gene Therapy Background and Research Motivation Charcot-Marie-Tooth disease (CMT) is a rare group of genetically heterogeneous neuromuscular disorders characterized by progressive muscle weakness and atrophy, along with sensory loss. Despite extensive preclinic...