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Pharmaceutical Targeting of Alport Syndrome Modifier Genes

Dr. Ron Korstanje
University of Maine
Alport Syndrome Foundation Grant
2019 - 2021
$125,000
Alport Syndrome

Lay Summary

Dr. Korstanje was awarded $125,000 over two years for his project which targets specific modifier genes that might reduce or delay the symptoms of Alport syndrome. There is large variation in the age of onset and severity of Alport syndrome in humans, even among patients with similar mutations. It is widely accepted that modifier genes contribute to this variation. Because modifier genes have been shown to be effective therapeutic targets in other diseases, they have potential to serve similarly in Alport syndrome. Using new mice models, Dr. Korstanje will determine if therapeutic intervention using a drug shown to reduce chronic kidney disease in other animal models could prolong kidney function.