WashU Nephrology congratulates Jeffrey Miner, PhD, FASN, who was awarded $132,000 from the Alport Syndrome Foundation (ASF). Miner, recently named Eduardo and Judith Slatopolsky Professor of Medicine in Nephrology, will use the grant to perform proof-of-concept gene therapy studies in a mouse model of Alport syndrome.
ASF is strategically investing in research that uses new technologies in the treatment of other diseases to potentially unlock a cure for Alport syndrome. The gene-editing tool CRISPR has attracted attention for its potential use as a curative therapy for monogenic diseases. Since Alport syndrome is the second most prevalent monogenic disease of the kidney, it needs to be determined if CRISPR will be a useful gene therapy treatment for Alport syndrome.
Miner, an internationally recognized expert on Alport Syndrome and Director of Basic Research for our division is part of a team of eight scientific researchers – the core of an inaugural Scientific Advisory Research Network (SARN)- assembled by ASF to devise a strategy to investigate whether CRISPR is a viable curative pathway.
The studies will begin with work in the Miner laboratory to determine the ages and stages of kidney disease at which promising gene therapy approaches, such as CRISPR, would be effective in Alport patients.
The ASF grant is funded through the generosity of patients, families and friends who donate to ASF.