A Wollongong professor was awarded almost $1 million to support her research into an incurable disease.
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Associate Professor Mirella Dottori, a researcher at the University of Wollongong will receive $982,861 over three years from the Medical Research Future Fund (MRFF) and National Health and Medical Research Council (NHMRC) for her ground-breaking research into Friedreich's ataxia.
Friedreich's ataxia (FA) is a rare genetically inherited disease that causes degeneration within the nervous system and heart tissue.
FA affects hundreds of Australians.
There is currently no cure and options to treat symptoms are limited.
"The cause of FA is mutations in a gene called Frataxin, which plays an important role in maintaining cell health," Associate Professor Dottori said.
"Individuals suffering from FA don't make enough Frataxin protein in their body, which impacts many tissues, particularly within the nervous system and heart."
The research being led by Associate Professor Dottori is a world-first approach to advance current technologies in gene therapy to treat FA.
"This research is using stem cells as a platform to develop an optimal gene therapy approach to treat FA," Associate Professor Dottori said.
"Gene therapy uses genetically modified and very safe viruses to deliver genes, proteins or drugs to the body as a therapy.
"We will use stem cells from FA patients to make nerves and heart cells 'in the dish', which will be used to screen gene therapy viruses to determine the optimal virus for delivering Frataxin protein."
The grant includes collaborators from the Children's Medical Research Institute at Westmead Hospital, Sydney, the Murdoch Children's Research Institute and St Vincent's Institute of Medical Research in Melbourne, the University of Melbourne and The Council of the Queensland Institute of Medical Research.
"This grant is the pinnacle of years of work from all of us bringing together our technologies and expertise to develop and deliver a potential curative therapy for FA," Associate Professor Dottori said.
While gene therapy clinical trials for treating FA have begun in the USA, the research team believe there are more efficient ways to do this, which would preserve other tissues during treatment - including the liver.
"Our aim is to develop an optimal gene therapy approach within the next 3 years, which will then be at the doorstep for clinical trials," Associate Professor Dottori said.
Professor Jennifer L Martin, UOW Deputy Vice-Chancellor (Research and Innovation), congratulated Associate Professor Dottori on receiving the grant.
"I am thrilled for Associate Professor Dottori and the broader research team," Professor Martin said.
"The award of this highly competitive Stem Cell Therapies Mission grant funding is testament to the quality of the interdisciplinary research, and to the impact the research outcomes will have on this devastating disease."
The research grant is being administered by NHMRC, on behalf of the Australian Government Department of Health, through the MRFF Stem Cell Therapies Mission grant.
The Stem Cell Therapies Mission supports world-leading stem cell research that develops and delivers innovative, safe and effective stem cell medicines to improve health outcomes that are accessible to all Australians who need them.
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