Award
NIH Reporter #5R01HD052682-15
Control of Breathing and Pompe Disease
Recipient
Not specified
Award Amount
$580,911.00
Ceiling
$580,911.00
Awarded
May 07, 2024
Identifier
5R01HD052682-15
Award for gene therapy research targeting Pompe disease to restore GAA expression and improve respiratory function, funded by NIH/NICHD.
Description
This project involves gene therapy research for Pompe disease, focusing on restoring GAA expression in the nervous system, skeletal, and cardiac muscles using AAV vectors. The project is funded by NIH/NICHD and aims to extend lifespan and prevent respiratory failure in affected models.