Two senior scientists at Children’s Medical Research Institute have been awarded prestigious Australian Government Medical Research Future Fund grants for their work in stem cells and genomics which will benefit patients with type 1 diabetes and a wide variety of inherited diseases.
The Stem Cell Medicine Group, led by Dr Anai Gonzalez Cordero, has been awarded $2.5 million to develop high-quality stem cells that will lay the foundation for Australia’s first cell therapies to treat vision loss in people with inherited diseases of the retina.
Dr Gonzalez Cordero’s work involves transforming small samples of skin or blood into stem cells – these are a form of early cell in human development that can turn into any cell type in the body. The work involves generating stem cell lines under laboratory conditions that are "clinical grade". These will become a rich source of cells for studying cell replacement therapies for retinal and other diseases.
“Our team is excited to be the first to develop these cell lines in Australia, to offer to researchers but also, most importantly, for the benefit of patients,’’ Dr Gonzalez Cordero said. “We want to give the same opportunities to Australians that are now available in a few places overseas.’’
Professor Sandra Cooper is an Adjunct Research Scientist at CMRI and Co-Head and Scientific Director of Kids Neuroscience Centre at the Sydney Children's Hospitals Network (Westmead). She has been awarded $2.9 million to integrate RNA testing into mainstream clinical practice.
For many individuals with genetic diseases it is still not possible to find the cause by sequencing the patient’s DNA, and for many of those undiagnosed patients the answer can be found by analysing the RNA. Professor Cooper’s ‘RNA for Rare Disease’ project involves a nationwide collaboration between research centres, pathology labs and clinical genetic departments to embed RNA Diagnostics as a new option for clinical diagnostics.
“We want to provide genetic answers for families who previously had none, enabling early diagnosis, early intervention, reproductive counselling, disease prevention and potential eligibility for relevant clinical trials,’’ Professor Cooper said. “With this grant we are now poised to translate RNA testing from a research context into practice. Australia will be among the first in the world to do so.’’
The CMRI researchers are all members of Luminesce Alliance, a cooperative joint venture established with the support of the NSW Government to improve children's health through world-leading research.