Award
National Institute of Diabetes and Digestive and Kidney Diseases 5R01DK131049-05
Restoring Ocrl1 function in Lowe Syndrome and Dent-2 disease
Recipient
Purdue University
Award Amount
$342,239.00
Ceiling
$342,239.00
Awarded
June 10, 2025
Identifier
5R01DK131049-05
This award funds research to restore Ocrl1 function in Lowe Syndrome and Dent-2 disease by testing candidate drugs and exploring innovative therapeutic approaches with high translational potential.
Description
The project focuses on testing candidate drugs, including FDA-approved compounds, to restore activity to Lowe syndrome protein variants. The project involves biochemical assays, kidney cell line studies, organoids, and zebrafish models. The project is innovative in proposing allosteric activators as a therapeutic approach. The project has high translational impact, potentially repurposing existing drugs for Lowe syndrome and Dent-2 disease.