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Article: Scientists at University of Western Australia publish research in muscular dystrophy.
- Article from:
- Biotech Week
- Article date:
- October 14, 2009
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According to recent research from Nedlands, Australia, "Duchenne muscular dystrophy (DMD), one of the most severe neuromuscular disorders of childhood, is caused by the absence of a functional dystrophin. Antisense oligomer (AO) induced exon skipping is being investigated to restore functional dystrophin expression in models of muscular dystrophy and DMD patients."
"One of the major challenges will be in the development of clinically relevant oligomers and exon skipping strategies to address many different mutations. Various models, including cell-free extracts, cells transfected with artificial constructs, or mice with a human transgene, have been proposed as ...