Article
Intravenous Administration of an AAV9 Vector Ubiquitously Expressing C1orf194 Gene Improved CMT-Like Neuropathy in C1orf194-/- Mice.
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics - 1 Oct 2023
Shen Zongrui, Li Meiyi, He Fei, Huang Cheng, Zheng Yingchun, Wang Zhikui, Ma Shunfei, Chen Li, Liu Zhengshan, Zheng Hui, Xiong Fu
Abstract excerpt
Charcot-Marie-Tooth (CMT) disease, also known as hereditary motor sensory neuropathy, is a group of rare genetically heterogenous diseases characterized by progressive muscle weakness and atrophy, along with sensory deficits. Despite extensive pre-clinical and clinical research, no FDA-approved therapy is available for any CMT type. We previously identified C1ORF194, a novel causative gene for CMT, and found that...
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