In a globally-significant step, Australian scientists have made a breakthrough that could lead to treatments for thousands of until-now untreatable inherited diseases.
The method has already been proven to stop the advance of one debilitating and ultimately fatal rare disease, a childhood muscle-wasting condition called Duchenne Muscular Dystrophy.
A Murdoch University team, which included Professor Steve Wilton, has developed two drugs, already approved in the United States, that exploit the cell machinery to trick cells into ‘skipping’ over the disease-causing error in the gene, acting as genetic ‘whiteout’.
“This is really the thin end of the wedge for this type of therapy,” Professor Wilton said.
“Depending on the type of mutation and the gene, we could theoretically treat and in some cases correct a variety of diseases that have previously been considered untreatable.”
“This type of precision medicine has the potential to transform healthcare on a scale equivalent to the way antibiotics transformed the fight against infectious diseases,” he said.
There are over 7000 inherited diseases and this type of therapy could be applied to many of them, depending on the type of mutation.
The team continues to work on treatments for spinal muscular atrophy, Marfan syndrome, Stargardt’s disease, epidermolysis bullosa, inherited blindness, cystic fibrosis, Pompe disease, Huntington’s disease and other types of muscular dystrophy.
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