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Potential Lifesaver Drug Announced

Can what is done be undone? In a part answer to that adage researchers have announced the developing //of a miracle drug to treat genetic disease- those which one is born with and which usually is not correctable.

Obviously the scientific world is too thrilled for silence. The drug named as PTC124 and been worked upon by researchers from PTC Therapeutics in New Jersey and those from University of Pennsylvania and Massachusetts , is slated to be licensed for public use by 2009.

The drug which trains the crosshairs on diseases caused by faulty genes, has been so far successful on Duchenne muscular dystrophy (DMD) and cystic fibrosis. The drug is being tested on humans now after promising results on mice and researchers hope it could eventually be used to treat sufferers of around 2,000 other types of hereditary disease.

DMD is known to afflict one in 3,500 boys and is marked by weakness and muscle wasting. Children born with cystic fibrosis suffer digestion and respiratory problems and tend to die early. Both these conditions are caused by so-called "nonsense mutations" in the child's DNA meaning that the sufferer lacks a vital protein.

Accordingly, in most genetic conditions, between 5-15 per cent of cases are caused by a defect called a “nonsense mutation”.

Genes are instruction manuals for cells to make proteins, but nonsense mutations in effect introduce a command halfway through that stops production. The kind of protein disrupted determines the nature of the disease.

In Duchenne muscular dystrophy, for example, the protein necessary for normal muscle development is not made, and the fatal wasting disease is the result. In hemophilia, it is the gene for the clotting agents factor VIII or factor IX that is disrupted.

Till now no known cure for either CF or DMD and sufferers rarely live beyond their 30s. Yet now the researchers have announced that mice suffering from a form of DMD began
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