Finding cures for children's genetic diseases


Rotary Club hear about latest research at CMRI


A senior researcher at Children's Medical Research Institute met with a community-based group that is contributing to his work this week.

CMRI’s Group Leader of Translational Vectorology, Dr Leszek Lisowski, spoke to the Hills Kellyville Rotary Club meeting on Monday night to talk about his work.

The club, as well as Australian Rotary Health and The FoxG1 Foundation, are funding research in Dr Lisowski’s lab. Hills resident Vivek Singha, who started The FoxG1 Foundation, is the father of five-year-old Kush. Kush has a mutation of the FoxG1 gene that has not been reported in any literature. He does not walk or talk and has suffered from severe seizures since he was very young.

Dr Lisowski’s work involves taking a virus, replacing all the negative elements of it and turning it into a “vector”, which is like a microscopic scalpel that can correct errors in DNA. This vector is then used to repair the mutation in the patient and ultimately provide a cure.

“At the moment the patient goes to hospital and finds out they have a mutation and they know everything about it, but the next question is – what do we do next?

“We are basically creating the surgeons and the surgical instruments of the future. We hope the next step will be creating a cure.’’

He explained that in the case of FoxG1 they need to target the central nervous system.

“The vector will have to correct the gene in the brain very specifically,’’ Dr Lisowski said.

“While this is driven by FoxG1 we hope it will have potential to target other conditions like Parkinson’s or Alzheimer’s. If it is successful, the impact will be much bigger than one disease.’’

Hills Kellyville Rotary thanked Dr Lisowski for attending to explain his work and invited him back for an update in the future.