50 Million euro investment for LUMC spin-off RougeTx advances therapies for inherited bleeding disorders.
Congratulations to reNEW Melbourne principal investigators Professor Andrew Elefanty and Associate Professor Richard Mills, and their teams based at Murdoch Children’s Research Institute, on being awarded National Health and Medical Research Council (NHMRC) Ideas grants.
The NHMRC Ideas Grants scheme supports talented researchers, at all careers stages and across all areas of research, to pursue creative approaches from discovery through to implementation, to address specific research questions.
Professor Andrew Elefanty and his team aim to correct mutations in stem cells from patients with inherited gene mutations that predispose them to developing leukaemia, in order to create personalised, rejection-free bone marrow stem cells.
“Our previous studies showed it’s possible to create blood stem cells in the laboratory that resemble those in the body. In this work we are further developing this method to make gene corrected blood stem cells that can be offered as a transformative new treatment for inherited blood disorders,” he said.
Professor Elefanty said he hoped the study would lead to future clinical trials.
Associate Professor Richard Mills and his team will test new treatments for the rare, degenerative muscle wasting disorder facioscapulohumeral muscular dystrophy (FSHD).
“We know the DUX4 protein drives skeletal muscle degeneration in FSHD,” he said.
“Our study will test 80 promising drugs in muscle tissue, grown in the lab from patient stem cells that express DUX4. This will get us one step closer to finding new treatments and improve the quality of life for patients.”
These grants recognise the strength of our research in tackling the biggest health challenges for Australians.
