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Muscular dystrophy treatment hope

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Treatment that may halt the progress of a fatal and incurable disease in boys may result from research at Royal Holloway, University of London.

Duchenne muscular dystrophy (DMD) is an inherited genetic condition that destroys muscles, and sufferers rarely live beyond the age of thirty.

The research, in collaboration with the MDEX Consortium and US biotechnology company AVI Biopharma, is into the anti-sense drug morpholino oligomer, also known as AVI- 4658.

It is a synthetic form of DNA which masks the corresponding portion of the patient’s gene, causing the damaged region to be bypassed, which stops muscle deterioration.

The drug relies on research into another rare form of MD in which the mutated gene is naturally in a masked form, which means that patients can lead a relatively normal life.

The Royal Holloway teams is led by Professor George Dickson, from the School of Biological Sciences.

Copyright Press Association 2009

Morpholino oligomer






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