Professor Steve Wilton
Professor Steve Wilton is the Director of the Perron Institute, Foundation Chair of Molecular Therapy at Murdoch University and Director of Murdoch University’s Centre for Molecular Medicine and Innovative Therapeutics (CMMIT). In collaboration with Professor Sue Fletcher, Professor Wilton has pioneered the use of antisense oligonucleotides (AOs) to overcome some of the most common dystrophin gene defects causing Duchenne muscular dystrophy (DMD).
Over a research journey spanning more than two decades, the Molecular Therapy Laboratory took the idea of using AOs to treat DMD from the laboratory to clinical application, through a partnership with the US pharmaceutical company, Sarepta Therapeutics (Cambridge, Ma). Three drugs for DMD have now been approved by the US Food and Drug Administration (FDA) and are relevant to nearly 30% of the DMD population. With co-leader Dr May Aung-Htut at Murdoch University, the Molecular Therapy laboratory is exploring therapeutic interventions for more than 50 different inherited and acquired conditions affecting human health. Professor Wilton has received multiple awards for his research on DMD and other diseases, including the Eureka Prize for Medical Research Translation, the Western Australian Innovator of the Year award and Officer of the Order of Australia.
Therapeutic alternative splicing: Gene patch strategies to treat inherited and acquired diseases