Healthier kids, brighter futures

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Help us accelerate our gene therapy research to the next level

Our Gene Therapy Unit is a joint initiative of CMRI and the Sydney Children’s Hospital Network. It is headed by Professor Ian Alexander, who is both a medical research scientist and a clinical paediatrician who treats sick children at the hospital. Ian and his laboratory team have developed a new therapy which cures genetic liver disease in mice. Now he wants to take a critically important step towards clinical trials of this therapy in children.

Why we need your help

  • The heartbreaking reality is that over a quarter of infant deaths are due to genetic disease. But now, for some of those babies, gene therapy brings real hope
  • Gene sequencing technology has provided unprecedented advances in understanding genetic diseases, but treatments now lag behind diagnostic powers. And that gap is growing rapidly
  • There will be children born in the future whose suffering will be avoided – genetic conditions that are incurable today will be treated and, in many cases, cured. For sick babies and their families, this will be life-changing
  • For their doctors, like Professor Alexander, it means fewer occasions of having to face the agonising duty of telling a parent that nothing can be done to help their child

Why we are excited about the future

  • Repairing and replacing faulty genes is not only possible, it’s happening today
  • Developing new therapies is the challenge for 21st century research. It is expensive, time-consuming and painstaking, but treatments must be found or children will continue to suffer
  • We have developed a treatment for genetic liver disease, and proved that it works in mice with these conditions. Now we need to modify the treatment to make it suitable for children
  • Please help us to get this new treatment ready to help children. Right now, lack of funds is the biggest barrier.  

Please donate today.

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