Published on November 16, 2021 | The PKD Foundation’s principal mission is to support basic, translational, and clinical research that will benefit patients with autosomal dominant and autosomal recessive polycystic kidney disease (ADPKD and ARPKD). This mission is strengthened by our international partnerships, including one with PKD Australia, whose co-funding with the PKD Foundation helps showcase to the world some of the most brilliant scientific minds in Australia. We’re pleased to share details of our most recent co-funded grant for Australian researcher, Melissa Little, Ph.D., who’s studying kidney organoids in ARPKD.
Melissa Little, Ph.D., Murdoch Children’s Research Institute
How did you first get involved in PKD research?
Melissa: My research has focused on kidney development and disease for several decades. It’s mostly focused on the molecular and cellular basis of kidney morphogenesis. We’ve been involved in identifying the role of new genes in this process. More recently, we’ve been able to use this understanding to build models of the human kidney from stem cells. It was then that we sought to investigate whether these stem cell-derived models were useful for understanding human disease, including polycystic kidney disease. We can achieve this by making stem cell lines from the patients and recreating a model of their disease in the laboratory.
What are you working on currently?
Melissa: Using stem cells, we’ve created a human collecting duct in a dish. This is the cell type in the kidney particularly affected in autosomal recessive PKD. Using a stem cell line with a patient mutation, we can show that the patient collecting duct differs from the control. We hope to use this as a tool to screen drugs to look for novel treatments for ARPKD.
What would you like the patient community to know about your research?
Melissa: Studies into inherited kidney diseases, such as PKD, are improving all the time. This provides patients with the hope of improved interventions that may save lives or improve their quality of life. This is our hope.
Do you have any personal connections to PKD?
What excites you most about this research?
Melissa: The possibility that we may be able to improve the lives of others.
What are some of your personal interests outside of research?
Melissa: My children and my dog. I like photography, painting, and being out in the wild.
Anything else you’d like to share?
Melissa: Our research is still a long way from providing the answers, but as well as now being able to better model this disease, one day we hope to also be able to generate replacement tissue for the treatment of disease.