A new drug has been discovered which offers hope to people who suffer from incurable genetic disorders such as muscle dystrophy and cystic fibrosis. 15% of genetic illnesses are caused by nonsense mutations in the genes. The drug PTC 124 binds to ribosomes in cells and allows nonsense mutations to be ignored during translation so that proper proteins can be made. The mutations are not corrected, just ignored so patients will have to take the drug for their whole life. The drug has been really successful in animal tests and has already passed human safety trials, there seem to be no side effects. The estimate is that it will be on the shelves in about 3 years.
This article is interesting because it relates closely to the areas of science we have been studying in BIOL 1020 and is an example of a real life application of this science. The drug can potentially cure any genetic disorder resulting from a nonsense mutation which means that over 1800 genetic diseases can potentially be cured.
http://www.timesonline.co.uk/tol/news/uk/science/article1690544.ece
