CORDIS Project
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This project aims to optimize gene therapy for Charcot-Marie-Tooth disease caused by mutations in glycyl-tRNA synthetase. By using viral vector-mediated gene transfer, it seeks to restore function in affected neurons and develop a business roadmap for further therapy development.
Charcot-Marie-Tooth (CMT) disease is the most common inherited neuromuscular disease, characterized by selective degeneration of peripheral motor and sensory neurons, leading to progressive muscle weakness and wasting, and sensory dysfunction.
Currently, no effective, FDA/EMA-approved therapies are available for CMT, necessitating life-long symptomatic treatment.
We recently identified the molecular mechanism underlying CMT caused by mutations in glycyl-tRNA synthetase (GlyRS).
We showed that tr…
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