Award
National Institute of Arthritis and Musculoskeletal and Skin Diseases 5R01AR082814-02
Expression of dystrophins with enhanced function
Recipient
University of Washington
Award Amount
$664,562.00
Ceiling
$664,562.00
Awarded
January 06, 2026
Identifier
5R01AR082814-02
NIH grant awarded to University of Washington for developing enhanced dystrophin gene therapies targeting Duchenne muscular dystrophy, focusing on novel AAV vectors and gene editing.
Description
This project aims to develop improved gene therapy vectors to deliver larger and more functional dystrophins, including full-length versions, using novel AAV technologies and gene editing methods. The research will focus on addressing current limitations such as vector size constraints, efficiency, and safety, with the goal of advancing therapies for Duchenne muscular dystrophy (DMD) and other neuromuscular disorders.