New Members June 2026
Hearing the words “no cure and no treatment’’ were devastating for mum Anna and that’s why she is asking people to donate to the gene therapy work being done at Westmead’s Children’s Medical Research Institute before June 30.
Anna’s daughter Adeline is six years old and lives with CDKL5 deficiency disorder, a rare and devastating neurological condition for which there is no approved treatment. She has lived with daily seizures for her whole life.
When she starting look around Australia for signs of hope, she found Professor Leszek Lisowski at Children’s Medical Research Institute who is working on a gene therapy for rare conditions like Adeline’s.
“Hearing the words “no cure” and “no targeted treatment” for your child is impossible to prepare for,’’ Anna said. “My daughter may never say “mum”, take her first steps, or look me in the eyes.’’
Adeline is the face of CMRI’s Tax Appeal and Anna is asking people to make a tax-free donation before June 30 to give families like hers more hope.
“While I can hold her, love her and fight for her every day, I cannot give her the answers that research has not yet found. At Children’s Medical Research Institute, researchers are working to better understand genetic disease and uncover treatments that could change the future for children like Adeline. “
Prof Lisowski has already helped develop groundbreaking gene therapy for other rare conditions and now wants to do the same for CDKL5.
“We want to get gene therapy for this disorder into development. Now that we’ve done it with a different condition, we have forged the pathway. We would be targeting the same organ, which is the brain, and using the same delivery vehicle.
“What we are doing with gene therapy is resetting the brain cells so they can start behaving normally. The child can start learning all over again.
“This research won’t be done without donations. We just can’t do it without that funding.”
By supporting this research, you are not only funding scientific discovery. You are giving families hope where none exists.
Donate now at https://myfundraiser.cmri.org.au/adelines-story
Children’s Medical Research Institute (CMRI) is a state-of-the-art medical research organisation dedicated to researching the genes and proteins important for human development, health and disease. Affiliated with the University of Sydney, CMRI is supported in part by its key fundraiser Jeans for Genes® and the Luminesce Alliance and is located at Westmead, a major health and innovation precinct in Sydney, NSW, Australia.
