Award
National Institute of Neurological Disorders and Stroke 5R01NS127403-04
CGT and ACD Inhibitors for SRT Treatment of Krabbe Disease
Recipient
Ernesto Roque Bongarzone and Jill A Morris
Award Amount
$589,359.00
Ceiling
$589,359.00
Awarded
July 25, 2025
Identifier
5R01NS127403-04
NIH grant awarded by NINDS for research on Krabbe disease using CGT and ACD inhibitors to test substrate reduction therapy combined with gene therapy and stem cell transplantation.
Description
This is a NIH grant opportunity for research on Krabbe disease involving the use of CGT and ACD inhibitors. The project aims to test the efficacy of substrate reduction therapy (SRT) combined with gene therapy and hematopoietic stem cell transplantation.