Award
Children's Hospital of Philadelphia 5R01NS126279-03
Network modulation to improve gene therapy in CLN3 disease
Recipient
Children's Hospital of Philadelphia, PA, United States
Award Amount
$487,607.00
Ceiling
$487,607.00
Awarded
August 05, 2025
Identifier
5R01NS126279-03
Award for research on improving gene therapy in CLN3 disease through network modulation, aiming to develop therapies for lysosomal storage disorders.
Description
This is a NIH grant project focused on gene therapy and network modulation in CLN3 disease, with a long-term goal to develop network-directed therapies to improve outcomes in lysosomal storage disorders.