Award
Eunice Kennedy Shriver National Institute of Child Health and Human Development 5R01HD052682-15
Control of Breathing and Pompe Disease
Recipient
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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 prevent respiratory and cardiac failure.
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 work aims to prevent respiratory and cardiac failure in early-onset and late-onset models.