University of Adelaide's Dr Rudrarup Bhattacharjee, Dr Adrienne Sullivan and Professor Jose Polo received funding from the MRFF for research into stem cell therapies.
A team of researchers from the University of Adelaide have received funding to help improve the lives of patients with rare neurodevelopmental disorders.
The team, led by Professor Jose Polo, Director of the Adelaide Centre for Epigenetics (ACE) and Program Leader at the South Australian immunoGENomics Cancer Institute (SAiGENCI), will receive $960,443 from the Medical Research Future Fund's 2024 Stem Cells Therapy Mission.
They will develop a comprehensive stem cell-based platform to study and find possible treatments for a range of rare conditions like Rubinstein-Taybi Syndrome, Wiedemann-Steiner Syndrome and Kabuki Syndrome.
"One of the biggest challenges in studying neurodevelopmental disorders is the lack of direct access to human brain tissue, making it difficult to model these diseases accurately," said Professor Polo.
"Stem cell technology allows us to reprogram skin cells of the patients into so-called induced pluripotent stem cells which can then be differentiated into any brain cell type, providing an unprecedented tool for studying these conditions and testing potential treatments."
Dr Adrienne Sullivan, Group Head of the Development and Epigenetics Lab within ACE and SAiGENCI, said the study will use CRISPR-based genome engineering and high-throughput drug screening to identify and trial potential therapeutics.
"These may alter the development trajectory of neurodevelopment disorders and may help enhance quality of life," she said.
Neurobiology postdoctoral research fellow in Prof Polo's group, Dr Rudrarup Bhattacharjee, said the complexity and rarity of NDDs make them difficult to study.
"Our approach will not only provide insights into disease mechanisms but also accelerate therapeutic discovery efforts to improve patient outcomes," said Dr Bhattacharjee.
Professor Anton Middelberg, University of Adelaide Deputy Vice-Chancellor (Research) congratulated Professor Polo on his grant.
"Professor Polo is an outstanding research leader conducting impactful team-based research," said Professor Middelberg.
"He is an incredibly valued mentor and member of our academic community, and this grant will allow him to advance his work in epigenetics towards new clinical interventions."
