Tuesday, April 24, 2007
New drug may cure genetic diseases - about time too !
The drug, called PTC124, will be revolutionary if it makes it all the way to chemists' shelves because it is a pill that could be used to correct aberrations programmed at a genetic level.
If its success is repeated in human trials, it could be useful to treat an estimated 15 per cent of patients suffering from up to 1800 different genetic diseases. Preliminary results, published online by Nature magazine, were encouraging.
New preclinical data published online in the current edition of the journal Nature show that PTC124, an investigational new drug designed to bypass nonsense mutations, was efficacious in a preclinical model of Duchenne muscular dystrophy (DMD). It is estimated that approximately 13 percent of the cases of DMD are due to nonsense mutations. PTC Therapeutics, Inc., which discovered and is developing PTC124, has catalogued over 1,800 distinct genetic disorders where nonsense mutations are the cause of the disease in a significant percentage of patients. Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70 percent of the individual cases of most inherited diseases, such as cystic fibrosis (10%) and Hurler's syndrome (70%).
IMO: For many, many years such successful gene therapy has been expected, so it is about time we had some results. Too much effort and money has been wasted instead on useless wars and terrorism, and overall both politicians and terrorists lose by their activities. Only a few cheap crooks and status-seekers like Tony Blair gain and important scientists don't get enough chances.
If its success is repeated in human trials, it could be useful to treat an estimated 15 per cent of patients suffering from up to 1800 different genetic diseases. Preliminary results, published online by Nature magazine, were encouraging.
New preclinical data published online in the current edition of the journal Nature show that PTC124, an investigational new drug designed to bypass nonsense mutations, was efficacious in a preclinical model of Duchenne muscular dystrophy (DMD). It is estimated that approximately 13 percent of the cases of DMD are due to nonsense mutations. PTC Therapeutics, Inc., which discovered and is developing PTC124, has catalogued over 1,800 distinct genetic disorders where nonsense mutations are the cause of the disease in a significant percentage of patients. Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70 percent of the individual cases of most inherited diseases, such as cystic fibrosis (10%) and Hurler's syndrome (70%).
IMO: For many, many years such successful gene therapy has been expected, so it is about time we had some results. Too much effort and money has been wasted instead on useless wars and terrorism, and overall both politicians and terrorists lose by their activities. Only a few cheap crooks and status-seekers like Tony Blair gain and important scientists don't get enough chances.
# posted by uv : 12:11 AM
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