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Child’s cells helping researchers develop customised treatments for inherited kidney disease

05 December 2018
Researchers model a patient's glomeruli to investigate kidney disease and test drug efficacy and toxicity.
3D modelling of a child’s kidney cells led by Murdoch Childrens Research Institute (MCRI) researchers is another step towards precision medicine, according to a paper published today in Nature Communications journal. 

Using kidney tissue created in an MCRI laboratory and induced pluripotent stem cells (iPSC) from a young patient the team, which included researchers from University of Melbourne, Royal Children’s Hospital and University of Manchester created a model of congenital nephrotic syndrome; an inherited condition that begins in infancy and typically leads to irreversible kidney failure. The research was led by Early Career Researcher Dr Lorna Hale. 

In modelling the condition, the researchers created a customised copy of the patient’s glomeruli, the part of the kidney’s waste filtration system damaged in this disease. The model enabled the researchers to better understand the cause of disease which they hope will ultimately lead to new treatments. 

Senior author, Professor Melissa Little, said these 3D models bring the potential to investigate normal kidney development as well as disease.

“These personalised models of the patient’s kidney will allow us to test drug efficacy and toxicity for individual patients in the future.” Professor Little said.

Precision medicine is set to transform Australia's healthcare and combines knowledge of a person’s unique genetic makeup, protein levels, and their environment to allow accurate disease prevention and treatment tailored to individual needs.

Professor Melissa Little is the Program Leader of Stem Cells Australia, and a Chief Investigator. 

Credit: Murdoch Childrens Research Institute

For more information:
Read the research article. 

Media Coverage
Herald Sun - "Kidney advance a step toward personalised medicine" - 6 Dec 2018.