£1.6m for research into a possible cure for muscular dystrophy

Mar 30 2004

The Health Secretary, John Reid, announced yesterday the successful bidders for £4m funding for research into gene therapy. £1.6m will go to the muscular dystrophy research group, which includes Dr Matthew Wood and Professor Kay Davies in the Department of Human Anatomy and Genetics at Oxford. The group is a consortium of leading UK scientists co-ordinated by the Muscular Dystrophy Campaign. Their new technique aims to place a 'patch' of DNA over the genetic errors which cause the disease.

Duchenne muscular dystrophy, a fatal muscle-wasting condition which reduces life expectancy to around 20, is the second most common genetic disease after cystic fibrosis, affecting one in 3,000 boys. It is caused by errors in the gene that causes dystrophin protein to be made. An error at one point along the gene causes the process by which the genetic code is converted to protein to be abruptly halted, meaning that production of dystrophin is stopped. Without the protein, muscle cells break down and die, causing disability and early death.

The DNA 'patch' will bind itself to the faulty DNA and act as a 'bridge' over it, so that the code instructing dystrophin protein to be made can continue to work. Rather than the process being halted altogether at the point where the error occurs, the inserted DNA will 'bridge' the faulty area, allowing the process to continue almost as normal.

The first stage of the project will involve human trials where the DNA 'patches' are inserted into single muscles to test for both safety and efficiency. While this is in progress Dr Wood and Professor Davies will investigate how the 'patches' can be delivered throughout the whole body. One possible method would involve attaching peptides to the DNA which could deliver the 'patch' preferentially to muscle cells.

Announcing the funding, John Reid said: 'I would like to particularly congratulate the Muscular Dystrophy Campaign for bringing together patients, parents, and scientists to address real needs and develop a novel treatment strategy for Duchenne muscular dystrophy. Their successful application for £1.6 million pays tribute to this outstanding collaborative effort, and should result in the first ever UK gene therapy trial for Duchenne muscular dystrophy.'

For more information on Duchenne muscular dystrophy go to the Muscular Dystrophy Campaign website.