Professor Steve Wilton
Steve Wilton

Professor Steve Wilton is the head of the Molecular Genetic Therapy Group at the Australian Neuromuscular Research Institute, University of Western Australia in Perth, Western Australia.  He obtained his PhD in Molecular Biology in 1983 in the Biochemistry Department, University of Adelaide, South Australia.  He was appointed Chief Production Biochemist in a University of Adelaide based biotech company, before being lured to Western Australia in 1987 to work for another biotech venture which supplied reagents for molecular biology research.  Becoming jaded with commercial production biochemistry, he started working after-hours with Nigel Laing at the Australian Neuromuscular Research Institute, before officially joining that institute in 1991.  He was involved in developing PCR based diagnostic tests for neuromuscular conditions, in particular pre-natal screening for Duchenne muscular dystrophy.

During studies of dystrophin gene transcripts from normal and dystrophic muscle, shorter than expected products were frequently detected, and these were found to arise from some naturally occurring exon skipping mechanism.  While presenting this work at a conference in Lake Tahoe, he met Prof Ryszard Kole (at that time at the University of North Carolina, Lineberger Comprehensive Cancer Center), who had been using antisense oligomers to correct abnormal splicing. After a true "Eureka moment", targeted exon skipping to compensate for protein truncating mutations in the dystrophin gene commenced in Australia. Two antisense oligomers were designed to the the exon carrying the mutation in the mdx mouse of muscular dystrophy…… and one worked!! We thought dystrophin exon skipping was going to be easy!!

Since that time, the Molecular Genetic Therapy Group, led by Professor Wilton and Associate Professor Sue Fletcher, have made substantial progress in the field of dystrophin exon skipping.  They were the first to show conclusive exon skipping in the mdx mouse model of muscular dystrophy, evaluated and identified oligomer chemistries that appear to have the greatest clinical potential (morpholinos), and have published a list of oligomers that can induce skipping of every human dystrophin exon from the mature transcript. Current work is focussing on refining oligomers for enhanced exon skipping efficiency to restore the reading frame in the most common deletion mutations, establishing efficient delivery regimens in animal models and establishing exon skipping strategies for non-deletion DMD cases.  The Wilton/Fletcher group is now extending exon skipping and splice intervention therapies to other genetic and acquired conditions.

Steve Wilton is also in charge of a high through-put genotyping facility (Molecular Genetic Research Services), which was established at the Institute in 1999. He is currently the vice-president of the Australian Gene Therapy Society, a member of Australian Society for Biochemistry and Molecular Biology, Genetics Society of Australia, Human Genetics Society of Australasia and the Lung Institute of Western Australia.  He enjoys drinking wine, cooking, watching bad movies (often at the same time), as well as fishing, scuba diving, wind-surfing and plans to age ungracefully.

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