Award
National Institute of Arthritis and Musculoskeletal and Skin Diseases 5P50AR065139-12
Overcoming limitations for AAV gene therapy
Recipient
University of Washington, Seattle, WA, United States
Award Amount
$670,615.00
Ceiling
$670,615.00
Awarded
August 18, 2025
Identifier
5P50AR065139-12
NIH funded project at University of Washington to improve AAV gene therapy for muscular dystrophies by enhancing vector potency and reducing immune responses.
Description
Gene therapy is a promising treatment for muscular dystrophies. The project aims to address current limitations of AAV gene therapies by testing new AAV capsid variants, micro-dystrophins, and vectors to minimize immunogenicity and enhance muscle potency, especially in cardiac tissue. It also involves applying enhanced gene silencing techniques for FSHD therapy.