Posted 22 June 2023

Gene editing could mean an end to inherited disease
Associate Professor Andrew Deans

Associate Professor Andrew Deans and his multidisciplinary team have been awarded Victorian Government funding to pioneer the development of RNA-based gene editing technology.

Using mRNA to deliver precise gene editing to bone marrow stem cells, a/Prof Deans is aiming to one day cure rare childhood diseases.

“Every year in Australia alone, more than 160 people are diagnosed with one of the many, often fatal, bone marrow failure diseases. Most them are young children and adolescents,” A/Prof Deans explains.

“At the moment the only treatment available is bone marrow transplant but, even if a compatible donor is found, transplant often comes with its own risks and complications. We’re aiming to use their own edited cells in order to tailor the treatment to the person.”

Although the technology is initially being developed to treat bone marrow failure syndromes, a/Prof Deans says it has enormous potential.

“Precise gene editing using RNA is the next step in treating inherited diseases,” he said. “We expect that this technology will eventually be used to treat any type of genetic disorder.”

Associate Professor Deans has been awarded $100,000 from the Victorian Government’s mRNA Research Acceleration Fund.