Kidney disease is often viewed as a disease of adults, but 1 in 15,000 children present with kidney disease as a result of inherited gene variants. Our research aims at increasing knowledge of genetic kidney disease in children and identify novel and effective therapeutic strategies.
Impact
Children with changes in specific genes can present with kidney failure in the first few years of life and no other treatment than dialysis or transplantation currently exists. By generating mini-kidney from patient-derived stem cells, we are able to improve our understanding of the cellular and molecular mechanisms driving the disease. This provides us with an opportunity to design therapies specifically correcting the defects identified.
reNEW research
Our team has found the underlying cause of one of the most common forms of genetic nephrotic syndrome. We have also developed a miniature stem cell-based model of the disease with which we will screen thousands of compounds looking for those able to correct the molecular problem.
Image description
A glomerulus in a stem cell-derived mini-kidney. The cells lighting up in the centre are podocytes, a key cell type for the filtration of the blood into the urine.
Credits
AI Dr Aude Dorison, Senior Research Officer, took the photograph. Ms Rachel Lam and Ms Irene Ghobrial prepared the sample. Prof Melissa Little Lab. reNEW Melbourne.
