Developing high content screens of novel treatments for congenital nephrotic syndrome

Summary Developing high content screens of novel treatments for congenital nephrotic syndrome

Project Details

Congenital nephrotic syndrome presents early in life and results in kidney failure and resulting severe proteinuria which can be life threatening. No treatments are available for this condition other than renal transplantation and dialysis. The genetically inherited forms of this condition most commonly result from mutation in genes expressed in the podocytes of the glomerulus. We have developed a method for generating human kidney tissue from pluripotent stem cells that represent good models of the human kidney. We have also established patient stem cell lines and gene edited stem cell lines with specific point mutations in genes known to result in congenital nephrotic syndrome.

This project would characterise these mutant kidney tissues and develop a high content screen to identify compounds that may be able to treat this disease. In this way, the objective would be to develop personalised treatments for congenital nephrotic syndrome.