The researchers intend to first demonstrate the utility of their new technology for gene therapy in Pompe disease, a rare genetic disorder that leads to progressive muscle weakness and damage to the heart.
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Centenary Institute receive funding for first therapy targeted at Pompe disease
Posted On: 30/06/2023
Researchers at the Centenary Institute and the Sydney Local Health District (SLHD) have been awarded $500,000 from CUREator, Australia’s national biotech incubator, to develop new technology to improve the safe and effective delivery of gene therapy.