Popping a pill could fix gene defect
11:03 17 October 03
Simply popping a pill might be enough to overcome the type of genetic defect responsible for many
cases of cystic fibrosis, muscular dystrophy and haemophilia. An experimental drug called PTC124
produces a working protein even though the gene remains defective.
Unlike gene therapy, it would work only for people with a particular kind of mutation. But while
gene therapists are still struggling to develop safe and effective ways of correcting genetic
faults, treatments like this could be available much sooner.
As many as a third of genetic diseases are caused by a premature stop mutation - a mutation in the
middle of a gene's sequence that stops the protein-making machinery at that spot. PTC124 binds to a
key component of this machinery, allowing it to read past the misplaced stop signal. As a result,
the full protein is made. Normal genes should not be affected, because their stop codon is at the
end of the messenger RNA strand rather in the middle.
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Comment: Artificially induced evolution follows? Or is the discovery of the process evolution
in itself?
Kind Regards, Robert Karl Stonjek.