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15 December, 2022

Ideas Grants awarded to four CMRI scientists

15 December, 2022

Ideas Grants awarded to four CMRI scientists


Research Leaders and senior postdoctoral scientists at Children’s Medical Research Institute (CMRI), located in the Westmead Health and Innovation District, have been awarded five grants as lead investigators in the highly competitive National Health and Medical Research Council Ideas Grant scheme for their work on genetic causes of blindness, and liver and kidney diseases. CMRI’s success rate for Ideas Grants was substantially higher than the national average. 

All of the CMRI investigators are affiliated with the University of Sydney and are members of Luminesce Alliance which supports advanced research into precision medicine for children.

Head of CMRI’s Translational Vectorology Research Unit, Associate Professor Leszek Lisowski, is Chief Investigator for two of the five successful research projects.


The first project, which also involves Dr Marti Cabanes Creus at CMRI and is a collaboration with Royal Prince Alfred Hospital in Sydney and the University of Navarra, Spain, aims to perform the first ever development and testing (validation) of advanced vector-based gene therapeutics using a whole human liver. The second project will involve working with the Centre for Eye Research Australia (CERA) in Melbourne and the University of Tasmania, to develop gene therapies for patients with inherited retinal diseases that cause blindness.

“We are very excited that the expert panels reviewing the grants recognised the importance and potential impact of both projects, which carry the promise of one day helping patients living with debilitating genetic diseases,’’ Associate Professor Lisowski said.

Dr Grant Logan and Dr Samantha Ginn, from CMRI’s Gene Therapy Research Unit, a joint initiative with the Sydney Children's Hospital Network, each received an Ideas Grant.


Dr Logan will develop novel methods for treating auto-immune kidney disease, working with co-investigator Dr Kim O’Sullivan from Monash University.

“This funding win is an incredible opportunity for us to advance development of a gene therapy approach to treat an auto-immune disease where the kidneys can be particularly affected,’’ Dr Logan said.

Dr Ginn will use gene therapy to target the devastating genetic liver disease called OTC Deficiency which can cause serious harm to children, including brain damage, and coma in the severest form which is life-threatening.

“This project will generate highly significant outcomes in the field of targeted liver gene therapy and provides a versatile platform for multiple liver diseases. For patients living with OTC Deficiency, this project offers the possibility of a permanent cure which is extremely exciting, It is a privilege to be working with a talented team of experts to make this happen,” said Dr Ginn.

SAm Ginn

Dr Ginn’s team includes Professor Ian Alexander as a chief investigator, and CMRI scientists, Associate Professor Lisowski and Dr Scott Cohen. Her work will also include collaborations with Professor Colin Pouton and Dr Harry Al-Wassiti from Monash Institute of Pharmaceutical Sciences; Professor Geoffrey McCaughan from Centenary Institute; Associate Professor Carlo Pulitano from Royal Prince Alfred Hospital and Professor Thomas Preiss from John Curtin School of Medical Research.

The fifth successful grant is led by Dr Anai Gonzalez Cordero, Head of CMRI’s Stem Cell Medicine Group. Her winning idea involves treating blinding eye diseases where the light-sensing cells of the eye (called photoreceptors) are defective, by developing replacement cell therapies. These are healthy photoreceptors generated in the lab, which will be transplanted into the eye with the aim of restoring function and treating/reversing blindness. A novel aspect of this work is that the team will test whether electrical stimulation of the healthy photoreceptors improves on the results – the idea being that this extra step will lead to positive changes to the nerve connections in the eye, enhancing the ability to restore vision.


“This is an ambitious project, but we are up for the challenge. My team and I are excited to conduct this novel research that tests new approaches. In the long-term it is patients who will benefit from this innovation,’’ said Dr Gonzalez Cordero

Showing efficacy for cell therapy will lead to faster translation of the research into clinical trials in patients.

The Director of CMRI, Professor Roger Reddel, noted that the outstanding strength of cell and gene therapy research at CMRI, which underpins all of these competitive grant successes, has been made possible by sustained, generous support from the Australian public through the Jeans for Genes Campaign. "This research will help position Australia among the global leaders in advanced therapeutics."