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College News
5 April 2004
Government funding for ground-breaking gene therapy
A consortium which includes Royal Holloway researchers has won £1.6m
in government funding for ground-breaking research into a possible cure
for muscular dystrophy.
The Health Secretary, John Reid, announced this week the successful bidders for £4m funding for research into gene therapy, of which £1.6m will go to the Muscular Dystrophy Research Group, which includes Professor George Dickson and Dr Ian Graham from the School of Biological Sciences at Royal Holloway, University of London. The consortium of leading UK scientists is co-ordinated by the Muscular Dystrophy Campaign, and includes research groups at Royal Holloway, Imperial, and the Universites of Oxford and Newcastle. Their new technique aims to place a DNA 'patch' over the genetic errors which cause the disease.
Announcing the Government 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."
Duchenne muscular dystrophy is a fatal muscle-wasting condition which reduces life expectancy to around 20 years. It is the second most common genetic disease after cystic fibrosis, affecting one in 3,000 boys, and is caused by faults in the gene that stimulates the production of dystrophin protein. An error at one point of the gene causes the process, in which the genetic code is converted to protein, to be abruptly halted, stopping also the production of dystrophin. And without the protein, muscle cells break down and die, causing disability and early death.
The DNA 'patch' aims to modify the faulty genetic code, by binding itself to the faulty DNA, and acting as a 'bridge' over the problem area. This will enable the code to continue with the production of dystrophin protein, without being halted at the point where the error occurs.
The first stage of the project will involve human trials, with the insertion of DNA 'patches' into single muscles to test for safety and efficiency. While this is in progress Professor Dickson and Dr Graham will investigate how the 'patches' can be optimised in terms of DNA sequence specificity and specialised chemical modifications to maximise the efficacy and delivery throughout the whole body.
Professor George Dickson said, "This new funding for clinical trials
is a vital step towards realising a safe and effective treatment for
Duchenne muscular dystrophy. Understanding this debilitating illness,
and finding a treatment, has been at the core of my scientific life
for over 15 years, and this particular gene therapy approach is based
on early pioneering experiments performed at Royal Holloway back in
the late 1990's. It is great to see a research idea coming to practical
fruition".
ENDS
For further information contact: Christine Long, Press & PR Officer, Tel: 01784 443967, email: christine.long@rhul.ac.uk
For more information on Duchenne muscular dystrophy visit the Muscular Dystrophy website www.muscular-dystrophy.org
Picture is available on request, please credit: Muscular Dystrophy Campaign
