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”
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”
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.
A gene therapy project to save infants’ lives has been named the top ranked National Health and Medical Research Council (NHMRC) Ideas Grant for 2020, in what the lead researcher describes as ‘proof that we weren’t just dreamers and symbolic of the power of the genomic revolution’. Professor Ian Alexander and his team were awardedContinue reading “Gene therapy project ranked top idea in Australia”
CMRI is pleased to announce that its partnership with LogicBio Therapeutics to develop the next generation of viral vectors for gene therapy applications has been extended for another two years and two new target tissues.
The Therapeutic Goods Administration (TGA) has registered a drug known as LUXTURNA® for the first gene therapy for inherited retinal diseases in Australia. One of our senior scientists, Professor Robyn Jamieson, has been involved in the process to get to this exciting stage.