Award
NIH Reporter #5R01NS127403-05
CGT and ACD Inhibitors for SRT Treatment of Krabbe Disease
Recipient
University of Illinois at Chicago
Award Amount
$589,115.00
Ceiling
$589,115.00
Awarded
August 14, 2026
Identifier
5R01NS127403-05
NIH grant awarded to University of Illinois at Chicago to develop combination therapies for Krabbe disease using inhibitors targeting psychosine synthesis, aiming to improve treatment outcomes for late-onset patients.
Description
This research aims to investigate new combination therapies for Krabbe disease, focusing on substrate reduction therapies (SRT) using compounds that inhibit ceramide galactosyltransferase (CGT) and acid ceramidase (ACD). The goal is to enhance existing treatments like hematopoietic stem cell transplantation (HSCT) and gene therapy, especially for juvenile and adult-onset patients.