A novel therapy for fatal diseases of the immune system 

At WEHI, we often reflect on how philanthropic support enables our early career researchers to be courageous with their research. It provides the opportunity for creativity and innovation, allowing them to develop their skills and progress their careers in medical research.

This is what occurred when The Jack Brockhoff Foundation provided an early career medical research grant to Dr Miles Horton to undertake research into gene therapy for immune diseases. This kind of opportunity is rare in that it provides a grant for a researcher to actively and independently explore their own original hypotheses, develop ideas, and test them.

Dr Horton’s research builds on a recently discovered novel gene editing technology that works as a spell check for human genes, called prime editing.

The Jack Brockhoff Foundation was established in 1979 by the late Sir Jack Brockhoff, who was committed to supporting medical research and other activities directed towards the health and wellbeing of children and young people. One of the ways the Foundation supports health and wellbeing is by providing grants to talented early-career medical researchers to strengthen research capability in Victoria. This in turn, encourages, facilitates, and accelerates their career paths to enable them to be competitive for additional funding support.

Support from The Jack Brockhoff Foundation has enabled Dr Horton to explore the potential of prime editing, which could pave the way to finding potential cures for immunological diseases such as fatal and severe genetic T-cell diseases, and other immunodeficiencies, that predominantly affect children and young adults. Dr Horton is using prime editing to perform highly targeted correction of DNA errors that cause these life-threatening diseases.

The key advantage of prime editing technology is that it can search for a specific sequence of DNA – where a disease-causing DNA error is located – and then precisely correct that error by replacing the incorrect DNA letter with the correct one. In doing so, prime editing functions like a spell check, searching the vast DNA genome for mistakes and precisely correcting them.

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Header image L-R: Dr Miles Horton and Mr Robert Symons, Chairman of the Jack Brockhoff Foundation.