Finding cures for children's genetic diseases

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HOPE FOR CHARLIZE

You can help us provide children with Propionic Acidemia and many other serious genetic conditions the opportunity to live disease-free, happy lives.


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“When you are told there is no treatment, no cure, all you want to do is change things for other people, so they don’t have to go through what we’ve gone through.’’

This is the vision of mother Julie Gravina who has already lost one child, Isaac, to a genetic condition. He required a liver transplant at the age of two but died soon after the risky operation. She is now doing all she can to ensure that his twin sister, Charlize, and future generations of children have a healthy future.

When Charlize had a liver transplant, Julie ensured that her liver cells were donated to Children’s Medical Research Institute for research into gene therapy.

With your help we have the opportunity to save precious little lives.


Professor Ian Alexander, who heads CMRI’s Gene Therapy Research Unit, and Dr Leszek Lisowski, who leads the Translational Vectorology Group, have established a dynamic research and translational collaboration. In the process, they’ve found unexpected synergies, resulting in a research effort that is much more than the sum of its parts. It has put them at the forefront of gene therapy globally.

It began with the creation of a world-first synthetic gene therapy vector, now in clinical trials to cure haemophilia, and has culminated in an ambitious strategy to tackle most genetic diseases, starting right here in Australia.

This technology could translate into saving the lives of infants with life-threatening conditions,’’ Prof Alexander said. “It’s about getting cures into the clinic as soon as we can.

 

“We’re trying to get to a point where instead of a liver transplant in a very young infant, we can genetically repair the liver without major surgery. It’s a very exciting time to be doing gene therapy.’’

As Julie says, “We look forward to a future where gene therapy will be available to completely cure our beautiful daughter, and children like her living with the most severe metabolic diseases.”

It is essential that we continue to work together to find cures for children affected by genetic diseases. Your motivation is clear as is ours – we all want to do everything we can to help ease suffering in children and their families.

Will you be a supporter of this research to help find cures for children’s genetic diseases?

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