The Manton Center for Orphan Disease Research
Jordan Kreidberg, MD, PhD
WT1 gene target discovery in FSGS
Focal Segmental Glomerulosclerosis (FSGS) is a severe condition leading to End Stage Renal Disease (ESRD), a condition in which an individual becomes dependent on dialysis or a kidney transplant, both of which are imperfect solutions accompanied by significant morbidity and mortality. The overall prevalence in the United States is estimated to be about 70,000 individuals, and there are approximately 20,000 people with ESRD due to FSGS. These include children and adults. Finding new treatments for FSGS is generally recognized as one the great challenges in medicine. Our studies are aimed at understanding the how kidney damage occurs in FSGS, with the ultimate goal that this research will translate into new treatments that prevent irreversible damage to kidneys and avoid the progression to dialysis and transplantation.