Molecular Therapies Research
The Molecular Genetic Therapies Research is led by Professor Steve Wilton and Dr May Aung-Htut and their laboratory is located at the Centre for Molecular Medicine and Therapeutics (CMMIT) at Murdoch University (a joint venture with the Perron Institute).
Their lab has a long history of cutting-edge research on novel genetic therapies for neuromuscular disorders, particularly Duchenne muscular dystrophy. In September 2016, the USA Food and Drug Administration (FDA) gave accelerated approval to a new treatment for Duchenne created by Professor Steve Wilton, Professor Sue Fletcher and their team. Eteplirsen (Exondys 51) is the first dystrophin restoring drug of its type ever approved by the FDA. In December 2019, the FDA granted accelerated approval for golodirsen (Vyondys 53), targeting another subset of Duchenne. In February 2021, the third FDA accelerated approval was granted for casimersen (Amondys 45). The three drugs developed by Professors Wilton and Fletcher and their team, can treat almost thirty per cent of patients diagnosed with Duchenne.
The main focus of the Molecular Therapies Research group is the use of small genetic ‘patches’ called antisense oligonucleotides (AOs) to mask part of a genetic message associated with a particular inherited disease. In the case of Duchenne muscular dystrophy (DMD), the defective genetic message is associated with the gene for the protein dystrophin, which plays a pivotal role in maintaining muscle structure and integrity.
- Molecular genetics
- Antisense oligonucleotide technologies
- Genetic therapies
- Exon skipping
- Splice switching
- Muscle repair and regeneration
- Inherited neuromuscular diseases
- Duchenne muscular dystrophy
- Spinal muscular atrophy