Muscular dystrophy gene therapy trial begins

20 October 2007

A new trial of prospective gene therapy to treat the genetic disease Duchenne muscular dystrophy (DMD) is about to begin in London. DMD is inherited in an X-linked recessive pattern, meaning that it affects predominantly males, around 1 in every 3500 live male births. The inherited mutation prevents the production of dystrophin protein, which leads to progressive muscle wasting beginning in early childhood. Affected children gradually lose mobility; the condition becomes increasingly serious as the wasting affects heart and respiratory function, leading to premature death by the early twenties.


There is as yet no effective treatment for the condition.It is hoped that by delivering a specially targeted genetic ‘patch’ in the form of antisense oligonucleotides, production of some functional dystrophin will be possible, and disease progression could be slowed. Animal trials of the drug have proved highly successful. Professor Francesco Muntoni of Imperial College London said: "Maybe this will not be a complete cure, but it could definitely buy a lot of time for these children" (see BBC news).


Funding for the trial was awarded by the  Department of Health in 2004, as part of the £50 million allocated in the 2003 Genetics White Paper (see previous news).

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