Running the red light

Traditional drug therapy for cystic fibrosis concentrate on the condition's symptoms, rather than its underlying cause - a faulty gene. A new drug, PTC124, tackles the gene in question not the symptoms. The drug aims to get the patient's cells to 'ignore' the genetic mutation that causes the disease. PTC124 is aimed at thwarting a cellular process called nonsense-mediated decay (NMD). NMD removes erroneous RNA molecules formed when certain mutations cause a stop-codon to be placed in the middle of a messenger RNA molecule. If these shortened versions of the cystic fibrosis protein were not destroyed by NMD they would provide some relief from the symptoms of the disease. This approach also holds hope for the treatment of other conditions such as beta-thalassemia.