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.
Muscular Dystrophy
Overview about muscular dystrophy, a muscle disease characterized by muscle weakness and muscle wasting.
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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.
![New Target for Muscular Dystrophy Drug Therapy Discovered]( "New Target for Muscular Dystrophy Drug Therapy Discovered")
Researchers at the University of Pennsylvania School of Medicine have discovered a new target for the muscular dystrophy drug therapy.
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
LIN/C
According to researchers, trials of the drug in mice have proved highly successful.
New Target for Muscular Dystrophy Drug Therapy Discovered
Researchers at the University of Pennsylvania School of Medicine have discovered a new target for the muscular dystrophy drug therapy.
Advertisement
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
LIN/C
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