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AAV9-mediated gene transfer of glycyl-tRNA as a novel therapeutic approach for GARS-associated Charcot-Marie-Tooth disease

assignment_turned_inproject01 Nov 2024(Started)-30 Apr 2025(Ended)Closed
Funder: Netherlands Organisation for Scientific Research (NWO)Project code: 21433
Funded under: Take-off WO fase 1 - Haalbaarheidsstudies 2024 TTW 1

AAV9-mediated gene transfer of glycyl-tRNA as a novel therapeutic approach for GARS-associated Charcot-Marie-Tooth disease

Description

Charcot-Marie-Tooth (CMT) disease is the most common inherited neuromuscular disorder for which an effective treatment is still lacking. We recently made a breakthrough by elucidating the molecular mechanism underlying a subtype of CMT (CMT2D) and demonstrating the therapeutic potential of an innovative treatment modality - tRNA. With this project we aim to show the feasibility of using a viral vector to deliver the tRNA molecule to peripheral nerves and to advance the development of a novel gene therapy for CMT.

Partners
Radboud Universiteit Nijmegen , Radboud Universiteit Nijmegen, Donders Institute - Donders Institute for Brain, Cognition and Behaviour, Centre for Brain, Cognition and Behaviour
Data Management Plans
argos Start a new DMP in Argos
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