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SEMINAR: Prospects for therapy of Duchenne muscular dystrophy

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Today's date is Friday, March 29, 2024
Prospects for therapy of Duchenne muscular dystrophy : School of Anatomy & Human Biology Seminar Series Other events...
The Seminar: Duchenne muscular dystrophy (DMD) is an X-linked muscle wasting disease caused by the absence of the 427-kDa cytoskeletal protein, dystrophin. There is currently no effective therapy. Recent advances in the understanding of the disease are leading to many different approaches to treatment which enhance the physiological performance of the muscle. Delivery of the dystrophin gene or minigenes using plasmids or viruses, exon skipping of mutations induced by oligonucleotides and readthrough of nonsense mutations are all moving into the clinic. We have focussed our studies on the development of a pharmacological approach which increases the levels of the dystrophin associated protein, utrophin. One small molecule was developed with Summit plc and was taken into a Phase I clnical trial by BioMarin Pharmaceuticals. This drug showed poor pharmakinetics but no toxicity and we have developed a novel mouse model to screen for follow-on compounds. We are also working on multi-exon skipping in the dystrophin gene using AAV develivery of antisense oligonucleotides. Such approaches to therapy hold great promise for the treatment of this devastating disease in the next decade.

The Speaker: Kay Davies is the Dr Lee's Professor of Anatomy at the University of Oxford and Head of the Department of Physiology, Anatomy and Genetics. Her research interests lie in the molecular analysis and development of treatment for human genetic disease, particularly, Duchenne muscular dystrophy, spinal muscular atrophy and ataxias. She is Director of the MRC Functional Genetics Unit aimed at exploiting genome information for gene function analysis in the nervous system. She has won numerous awards for her work and also has an interest in the ethical issues associated with genetics research. She is a founding fellow of the UK Academy of Medical Sciences and was elected a Fellow of the Royal Society in 2003. She has been a Governor of the Wellcome Trust since 2008 and was made Dame Commander of the British Empire for services to science in 2008.
Speaker(s) Professor Dame Kay Davies, Dr Lee�s Professor of Anatomy, Director, MRC Functional Genomics Unit, Head of Department,Physiology Anatomy and Genetics, University of Oxford, UK
Location Anatomy & Human Biology, 1st Floor Seminar Room (1.81)
Contact Debbie Hull <[email protected]> : 6488 3290
URL http://www.anhb.uwa.edu.au/about/seminar_program/CurrentSeminars
Start Tue, 03 May 2011 13:00
End Tue, 03 May 2011 14:00
Submitted by Debbie Hull <[email protected]>
Last Updated Thu, 17 Mar 2011 13:58
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