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 will 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…
