Drug Targets Hundreds of Disorders

Magic bullet hits mutations that prompt 1,800 genetic diseases
By Peter Fearon,  Newser Staff
Posted Apr 23, 2007 8:42 AM CDT
A lab officer cuts a DNA fragment under UV light from an agarose gel for DNA sequencing as part of research to determine genetic mutation in a blood cancer patient, Thursday April 19, 2007 in Singapore,...   (Associated Press)

(Newser) – A magic bullet that could treat cystic fibrosis, muscular dystrophy, hemophilia and more than 1,800 other genetic disorders could be available by 2009. Lee Sweeney of UPenn, leader of the team developing the drug, tells the Times of London: “It doesn’t just target one mutation that causes disease, but a whole class of mutations.”

The breakthrough drug, known as PTC124, works by helping genes to ignore disruptions to genetic code caused by "nonsense mutations," allowing cells to develop normally. It can be used against a wide range of disorders caused by the same kind of mutation. The daily pill enters the last phase of trials on humans this year.