Award
National Institute of Neurological Disorders and Stroke 5R61NS136651-02
tRNA Gene Replacement Therapy for tRNA-Synthetase Related Charcot-Marie-Tooth Disorder
Recipient
Jackson Laboratory, Bar Harbor, ME, United States
Award Amount
$411,015.00
Ceiling
$411,015.00
Awarded
June 05, 2025
Identifier
5R61NS136651-02
Award for research on gene therapy targeting Charcot-Marie-Tooth disease type 2D using AAV9 vectors to deliver tRNAGly genes, aiming to develop a new therapeutic approach.
Description
This project focuses on developing gene therapy using AAV9 vectors to deliver specific tRNAGly genes to treat Charcot-Marie-Tooth disease type 2D caused by dominant mutations in the GARS gene. The research includes constructing vectors with multiple tRNAGly genes, comparing different capsids for systemic delivery, and testing efficacy and durability in mouse models.