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Drugs, Vaccines and Therapeutics
, Gene Therapy
by ruth on April 25, 2007
Scientists report of a new prototype drug, designed to treat Duchenne muscular dystrophy but which may be useful in the treatment of up 1800 different genetic diseases. The drug, called PTC124, has restored muscle function in mice trials.Normal cells assemble amino acids into protein molecules essential for good health according to a "recipe" encoded into the DNA. In about 15 per cent of patients with DMD, a DNA "nonsense mutation" causes the cells to stop making the protein chains prematurely, rendering the proteins useless.
PTC124 fixes this by prompting the cells to ignore the stop mutations that tell them to end the protein chain, allowing themto complete the correct sequence.
Human trials of PTC124 are being conducted for DMD and cystic fibrosis. The authors of the study published this week in Nature, may also have potential in treating other genetic diseases due nonsense mutations.
See the full report from the Herald Sun or read the Nature manuscript, "PTC124 targets genetic disorders caused by nonsense mutations" (abstract free).
Tags:
genetic+disease
Duchenne
muscular+dystrophy
genetics
biotech
duchenne+muscular
genetic+diseases
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Vote for PTC124 Drug Candidate Against Duchenne Muscular Dystrophy and Other Genetic Diseases:
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Rating: 7.71 out of 7 vote(s) cast.
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Response from:
Arun
(12/07/07 5:59am)
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This mail is being sent from India.
I am 49yrs male suffering from limb-girdle muscular dstrophy as diagonised at
CMC -Vellore on advice of prof.John Walton of UK.My sister aged 47 is also
suffering from MD.
I came to know about PTC124 which is a remarkable step forward towards the
treatment of MD and want to know latest developments in this regard.
i want to offer myself as a volunteer for medical trial if it is possible from
your side.Iwould also like to know if there has been any such trials earlier
and if there was any positive result of the same.supply of any medication which
would provide some relief of any sort would be possible.
Any other information in this regard will be highly appreciated by me.
hoping to have your reply at the earliest,
with regards,
Arun K Gheedia.