Children’s Medical Research Institute (CMRI) is excited to announce a major grant by Cure4 Cystic Fibrosis Foundation awarded to a team of our scientists.

The Foundation announced today that its Holckner Family CF Impact Grant has been awarded to a team from CMRI and The University of Sydney, including Associate Professor Leszek Lisowski (Head of CMRI’s Translational Vectorology Unit), Dr Andrea Perez-Iturralde, Ms Kimberley Dilworth, and Professor Hiran Selvadurai from Children’s Hospital Westmead.

This is the second major grant to CMRI researchers from Cure4 Cystic Fibrosis Foundation, thanks to fundraising efforts of the Team Simon Foundation who have a long history with CMRI.

Associate Professor Lisowski thanked both foundations.

“We are very honoured to have been selected by Cure4CF to join their amazing team, and the other research teams they support, in the fight to bring a cure to the many children suffering from CF,’’ he said.

“When it comes to developing a cure for a devastating disease like CF, it takes three critical components: dedication, money, and the team. We have got the dedication and now thanks to Cure4CF we also have the very much needed financial means to push the research forward. But the most important thing is that by joining forces with Cure4CF, we now have an unstoppable team working closely together. We hope that for children born with CF, our dream of a CF-free future, will one day become a reality.’’

Thanks to the previous Cure4 Cystic Foundation grant, Associate Professor Lisowski’s team is two years into the work, which aims to use gene therapy to correct CF-causing mutations in the CFTR gene. They hope to deliver a universal treatment which changes the person’s non-working gene to allow it to work properly.

Dr Andrea Perez-Iturralde said they hoped that by the end of this project they would be able to begin studies to inform a clinical dosing and delivery strategy conducted by clinical colleagues at the children’s hospital.

“Being chosen by Cure4CF to contribute to the battle against CF is an immense honour. My deep concern for the impact of incurable disorders, particularly on children, fuels my strong motivation to make a meaningful difference,’’ she said.

“After working with Leszek on a project that is currently progressing towards clinical applications, I am eager to replicate that success in the realm of cystic fibrosis.

“Thanks to the support from Cure4CF, we've been empowered with the funding needed to pursue essential research, and our goal is to reciprocate on this support by developing an effective strategy for treating CF.’’