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Muscular Dystrophy To Be Targeted With Gene Therapy : UK Trials

by Medindia Content Team on October 22, 2007 at 5:50 PM
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Muscular  Dystrophy To Be Targeted With Gene Therapy : UK Trials

British researchers are going to start a gene therapy trial in London for the fatal disorder Duchenne muscular dystrophy (DMD).

In the first such trial in the world, a small group of patients will be injected with an experimental drug which might extend their lives, the BBC reported Friday.


DMD is caused by a single faulty gene and results in progressive muscle wasting. The new treatment involves the making of a very small piece of genetic material -- a molecular patch, which once inside the muscle cell, corrects the genetic error and makes the gene work again.

The treatment enables the gene to produce a working version of the dystrophic gene, which keeps muscles strong.

According to researchers, trials of the drug in mice have proved highly successful.

At first, minute quantities of the drug will be used to check its safety. If it works, the drug will effectively knit together the key damaged section of DNA, allowing it to begin producing a protein that keeps the muscles strong.

The researchers said if it works in humans, patients would need regular infusions of the drug.

Lead researcher Francesco Muntoni from Imperial College London has high hopes that it could slow or even halt the progression of muscle wasting and give some patients the chance of living into old age.

Muntoni describes the gene therapy as like a piece of molecular Velcro, which will make a temporary repair.

Source: IANS


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