Several Children’s Medical Research Institute (CMRI) scientists and their teams have been awarded highly competitive National Health and Medical Research Council Ideas Grants.
Professor Tracy Bryan, Head of CMRI’s Cell Biology Unit and awardee in this round, will use the grant to study a gene-editing strategy for telomere-related bone marrow failure. For patients with bone marrow failure, the only treatment available currently is a bone marrow transplant. Professor Bryan believes these patients would be ideal candidates for gene therapy.
“We have teamed up with some of the international leaders in development of gene editing technology with the goal of providing the first preclinical demonstration of the effectiveness of gene therapy for telomere biology disorders,’’ Professor Bryan said.
She will be collaborating with other CMRI researchers working in the areas of gene therapy and bone marrow disorders, including Professor Ian Alexander, Associate Professor Leszek Lisowski, and Associate Professor Karen MacKenzie.
Professor Robyn Jamieson, Head of CMRI’s Eye Genetics Research Unit, was awarded a grant for her work on ALPK1 dysfunction, studying this novel pathogenic (disease-causing) pathway to help treat retinal (eye) diseases that cause blindness. Professor Jamieson is aiming to create a new therapy for inherited retinal diseases, as well as other ocular and inflammatory disorders.
“Demonstration of therapeutic benefit of these approaches will provide valuable opportunities to develop new treatments.’’
Professor Jamieson will work with several other CMRI researchers and teams, including Dr Mark Graham, Dr Anai Gonzalez Cordero, Dr Pengyi Yang, and Professor Ian Alexander.
Dr Wendy Gold, who works across both CMRI and Kids Research, was part of a team that was awarded an Ideas Grant for work on accelerating treatment for epilepsy in children with KIF1A-associated neurological disorders.
CMRI teams and facilities including those led by Dr Anai Gonzalez Cordero and Dr Mark Graham will be involved in Dr Gold’s grant-funded research.
CMRI’s Director Professor Roger Reddel congratulated the teams for their success in an extraordinarily competitive environment.
“All of these researchers are outstanding in their field. This has been recognised by their scientific peers through these awards, which are testament to bold ideas and vision, and the strength of collaboration across many CMRI research teams – designed to deliver significant impact for patients’’ Professor Reddel said. “We are grateful to NHMRC for supporting this vision.’’