Developing high content screens of novel treatments for congenital nephrotic syndrome

Research Opportunity
PhD students
Department / Centre
Paediatrics
Location
Royal Children’s Hospital/Murdoch Childrens Research Institute
Primary Supervisor Email Number Webpage
Prof Melissa Little melissa.little@mcri.edu.au Personal web page
Co-supervisor Email Number Webpage
Dr Aude Dorison aude.dorison@mcri.edu.au

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.



Faculty Research Themes

Child Health

School Research Themes

Child Health in Medicine



Research Opportunities

PhD students
Students who are interested in joining this project will need to consider their elegibility as well as other requirements before contacting the supervisor of this research

Graduate Research application

Honours application

Key Contact

For further information about this research, please contact a supervisor.

Department / Centre

Paediatrics

Research Node

Royal Children’s Hospital/Murdoch Childrens Research Institute

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