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Novel protein replacement therapy to combat incurable genetic disorders

24 months
Approved budget:
Associate Professor Geoffrey Krissansen
Health issue:
Human genetics and inherited/congenital conditions
Proposal type:
Explorer Grant
Lay summary
There is no cure for the majority of hereditary genetic disorders arising from single gene defects. Two of the cruellest such diseases are cystic fibrosis and hereditary diffuse gastric cancer, which are associated with high morbidity and lead to premature death. Here we propose to employ novel drug delivery technology to replace the defective proteins associated with each disease to provide a potential cure. The technology has the potential to transform medical practice in the prevention or treatment of genetic disorders that are currently incurable. The provision of a cure for an incurable disease is transformative, with the potential for a major impact on the lives of those affected.