Award
National Institute of Neurological Disorders and Stroke 5R01NS126279-04
Network modulation to improve gene therapy in CLN3 disease
Recipient
Children's Hospital of Philadelphia
Award Amount
$487,607.00
Ceiling
$487,607.00
Awarded
July 27, 2026
Identifier
5R01NS126279-04
NIH awarded a grant to Children's Hospital of Philadelphia for research on improving gene therapy in CLN3 disease by studying network modulation and therapeutic windows for gene replacement.
Description
This NIH grant project focuses on gene therapy and neurodevelopmental research in CLN3 disease. The project explores the therapeutic window for gene replacement and network modulation, aiming to develop combined therapies to improve outcomes in lysosomal storage disorders.