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The Future of Medicine

Our vision is to continue to drive the development and accelerate the delivery of world-leading gene and gene-modified cell therapies to improve the health of all Australians.

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Australian first gene therapy for childhood blindness

Two Sydney siblings have become the first patients in the country to receive a novel gene therapy that has rescued their vision and holds hope for preventing them from going blind. The ocular gene therapy, LUXTURNA, is the world’s first approved gene replacement therapy for an inherited blinding eye condition and one of the firstContinue reading “Australian first gene therapy for childhood blindness”

CMRI Awarded Multiple Medical Research Future Fund Grants

Children’s Medical Research Institute (CMRI) was awarded multiple Medical Research Future Fund (MRFF) grants to help improve the lives of children living with genetic diseases. The MRFF, which is an initiative of the Australian Government, has funded research projects in cancer, gene therapy, and stem cell medicine at CMRI. Dr Anai Gonzalez-Cordero, head of theContinue reading “CMRI Awarded Multiple Medical Research Future Fund Grants”

Gene therapy trial success: game changer for children with SMA

New research has shown that gene therapy may provide an effective treatment for Spinal Muscular Atrophy (SMA), a devastating and fatal genetic condition. SMA affects the motor nerve cells in the spinal cord, causing progressive muscle weakness and preventing babies from being able to roll, sit up, crawl, walk and eventually breathe. Until recently, it was the leading genetic cause of infant death in Australia, occurring in 1 in every 10,000 births.

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Australian Genome Therapeutics Centre is a joint initiative of