SEMINAR: �Antisense oligomer therapies for neuromuscular disorders�

Sue Fletcher completed her first degree at the University of Zimbabwe (when the country still had a functional education system) and a PhD at UWA. She is a Principal Research Fellow working with Steve Wilton on developing antisense therapies for inherited disorders at the Centre for Neuromuscular and Neurological Disorders, University of Western Australia. The Wilton laboratory is located within the Australian Neuromuscular Research Institute, which is also the home of Muscular Dystrophy Western Australia. This group pioneered antisense oligomer induced exon skipping to overcome dystrophin mutations, and have developed antisense oligos for every dystrophin exon, except the first and last. Although not all dystrophin mutations are amenable to exon skipping intervention, and the potential improvements will depend upon the nature and location of the mutation, oligomer strategies should be developed for all, not just those with common mutations. The group are now exploring ways to overcome DMD-causing non-deletion mutations, including duplications and extending the technology to other inherited neuromuscular disorders, including spinal muscular atrophy.