Molecular Genetic Therapy Research

The Perron Institute’s Molecular Genetic Therapy Research led by Professors Steve Wilton and Sue Fletcher at Murdoch University has a long history of cutting-edge research on novel genetic therapies for neuromuscular disorders, particularly Duchenne muscular dystrophy.

Their exon skipping drug Exondys 51 (eteplirsen) received accelerated approval by the US Food and Drug Administration (FDA) in 2016.
The main focus of their research is the use of small genetic ‘patches’ called antisense oligonucleotides (AOs) to mask part of a genetic message associated with a particular inherited disease. MORE