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Royal Holloway to develop treatment for life-threatening genetic muscle disease

Posted on 02/11/2011

Professor George Dickson

Royal Holloway, University of London has signed an agreement with Benitec, an international  biotechnology company focused on developing methods to treat serious diseases, to begin a new program to develop a novel therapy and potential cure for an untreatable, debilitating and potentially life threatening genetic muscle disease, oculopharyngeal muscular dystrophy (OPMD).

The research will be carried out under the direction of Professor George Dickson, from the School of Biological Sciences, at Royal Holloway and in laboratories in Paris, by Dr Capucine Trollet at the Institut de Myologie, in the Universite de Pierre et Marie Curie (UPMC).

OPMD is an inherited, slow progressing, degenerative muscle disorder that prevents the patient from swallowing, often leading to death by choking. It is caused by a genetic mutation that destroys the swallowing muscles.

There is currently no cure or medical treatment available for OPMD. Symptomatic surgical interventions are used to temporarily improve swallowing in moderate to severely affected individuals, but these are inadequate treatments for most afflicted individuals.

OPMD is a rare disease (1/100 000 in Europe), however it is the most common muscular dystrophy in Quebec, Canada (1/1000).

The aim of the collaboration is to develop a ddRNAi interference-based therapy, which is one of two major approaches to RNA interference - which is a system within living cells that takes part in controlling which genes are active and how active they are. The idea is to use this approach to silence the expression of the mutant gene in muscle cells of OPMD patients. OPMD is particularly adapted to gene therapy since the affected cells are limited, the genetic mutation is small, known, and located on a relatively small gene. 

Professor Dickson said: “The opportunity to work with Benitec and to access their impressive patent position in gene silencing is very exciting for us. We believe that Benitec’s technology can lead to a breakthrough treatment, and even a cure, for this rare but often fatal disease. We are looking forward to making this program a clinical reality utilising the wonderful team and resources at the Royal Holloway University and at the Institut de Myologie.”

Dr French, Benitec CEO adds: “Benitec’s strategy is to demonstrate the power and the potential of its RNA interference technology across a range of severe life threatening diseases, and the OPMD program extends our reach into treating and curing genetic disorders in addition to our cancer and infectious disease programs, Professor Dickson and Dr Trollet will join Benitec’s Chief Investigators Group and, as international experts in genetic disease and RNAi, will contribute significantly to Benitec’s intellectual capital and reputation for scientific and clinical excellence.”



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