Children's Medical Research Institute is Australia’s oldest paediatric research organisation and has had global impact. We helped eliminate Rubella, introduced care for premature infants, and developed microsurgery techniques for organ transplants that save children’s lives every day.
1 in 20 kids face a birth defect or genetic disease, and 30% of children’s hospital admissions are due to genetic disorders, so we know there is still much work to be done.
These kids and their families are used to hearing words like “incurable” or “lifelong effects”. It doesn’t have to be this way. Right now, we’re working on gene therapy to find cures for previously incurable genetic diseases.
A genetic disease is a serious condition caused, at least in part, by changes in our DNA. While often inherited, many, like cancer, happen ‘out of the blue’ due to chance occurrences. There are more than 6000 different genetic diseases. Most are poorly understood and have no specific treatments or cures.
Gene therapy is a method of curing genetic diseases by correcting the cause, by fixing an error in our DNA. Our researchers have helped deliver gene therapy cures in Australia for boy in the bubble disease, spinal muscular atrophy, and the cure we’ve developed for metabolic liver disease is entering clinical trials in the UK.
Right now, we are establishing a new gene therapy initiative to fill the gap in Australian medicine. We want to ensure children facing genetic diseases get more than a diagnosis—they get a cure, one that’s available right here at home.
No child’s life should be cut short, and with your support we can change “incurable” to “curable”.