Funding boost brings new hope for rare disease sufferers

November 28, 2017

NHMRC funding: Professors Steve Wilton and Sue Fletcher will work to develop genetic therapies for rare diseases.

Researchers at Murdoch University have been awarded significant federal funding to help develop genetic therapies for rare diseases.

Professor Steve Wilton will lead the project, which has received $800,000 from the Australian Government’s National Health and Medical Research Council (NHMRC) over the next three years.

This project will build on two decades of research by Professor Wilton and Professor Sue Fletcher that has resulted in the first ever treatment to have altered the progression of the fatal disease Duchenne Muscular Dystrophy (DMD).

Professor Fletcher explained the technology.

“We have exploited the fact that some genes linked to inherited diseases have sections that are potentially dispensable,” Professor Fletcher said.

“Our technology, known as exon skipping, acts as a genetic whiteout that tricks cells into skipping over a genetic mutation.

“This has resulted in a far better functionality of the dystrophin protein and improved function in DMD, and we believe these strategies can be applied to some other genetic diseases.”

Rare diseases affect around one in 12 Australians, but there are over 6,000 known rare diseases, which means often there are only a handful of men and women with a disorder.

Over the next three years the research team will focus initially on eight genes that contribute to 46 serious inherited disorders.

“This technology provides an exciting new platform to develop targeted therapies for a host of rare diseases that currently do not have successful treatments,” Professor Fletcher said.

“We anticipate that several potentially therapeutic compounds will be developed during the course of this project.”

The Murdoch University team will work with long-term collaborators at UWA and Orthocell to develop the new therapies.

MurdochUniversity is also involved in six successful projects totalling over $5.5m led by UWA, TKI, Griffith University and Curtin University.

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