Institute of Advanced Studies

Approaches to muscular dystrophy therapy: the partnership between patients, clinicians and biotechnology

Professor Dame Kay Davies will discuss the recent advances in the field of muscular dystrophy research and will explain how some of the therapeutic approaches being studied by scientists, either separately or in combination, hold great promise for the treatment of these diseases in the next decade.

Human muscle diseases can be very devastating, as they are often clinically severe, with very little available in the way of treatment.  The most commonly inherited muscle disease of childhood is named "Duchenne Muscular Dystrophy" and it affects mainly males due to the causative gene, dystrophin, being located on the X chromosome.  Since the identification of the defective gene in 1986, much hope has existed for the development of a therapy and indeed a cure for this disease.  Additionally since this time, the causes of many other forms of muscular dystrophy have been found.  Despite over 20 years of exhaustive research focussed towards establishing cures, no treatment based on the knowledge of causative genes is currently in use, although a significant amount of knowledge has been created about how these diseases manifest.

This area of research is currently going through an exciting era however, with a number of human clinical trials in a variety of therapeutic approaches having just been conducted, or presently being performed.  Some of the exciting approaches being investigated include stem cell therapy, gene therapy replacement of the missing gene using viruses or plasmids, correction of the defective gene, switching on other proteins as substitutes for the defective or missing ones among others. 

Biographical details

Kay Davies is Dr Lees Professor of Anatomy at the University of Oxford and Head of the Department of Physiology, Anatomy and Genetics. She is Director of the MRC Functional Genomics Unit whose aim is to exploit genome information for the analysis of the function of genes in the nervous system with a view to determining the molecular pathways involved in movement and behavioural disorders.

This free lecture is part of the Indian Ocean Rim Muscle Colloquium being held on Wednesday 21st - Friday 23rd January 2009, at The University of Western Australia, Perth, Western Australia.