Award
NIH Reporter #5R01HD052682-15
Control of Breathing and Pompe Disease
Recipient
University of Florida, Gainesville, FL
Award Amount
$580,911.00
Ceiling
$580,911.00
Awarded
May 07, 2024
Identifier
5R01HD052682-15
A gene therapy research project funded by NICHD to develop and evaluate AAV gene therapy approaches for Pompe disease using novel rat models, aiming to prevent respiratory and cardiac failure.
Description
This project focuses on gene therapy for Pompe disease, aiming to improve treatment outcomes and contribute to the broader field of neurodegenerative and autosomal recessive diseases. It uses rat models to test adeno-associated virus (AAV) gene therapy to restore life-long GAA expression in nervous system, skeletal and cardiac muscle to preserve cardiorespiratory function and prolong life.