Scientists using genetics to unravel the molecular basis of a genetic disorder that can cause skeletal malformations and heart abnormalities have discovered that a widely used hypertension drug prevents a deadly complication of the disorder in animals.
A team of scientists at Johns Hopkins University has found that treatment with losartan prevented the development of aortic aneurysm and other defects in a mouse model of Marfan syndrome (Habashi JP et al. Science. 2006;312:117-121). Based on these promising findings and losartan's established safety in humans, the investigators plan to launch clinical trials of the drug as a treatment for the disorder in the fall.
The 1991 discovery that mutations in the gene that encodes fibrillin-1 cause Marfan syndrome and deliberations about how these mutations might lead to the problems seen in patients with the disorder led researchers to this breakthrough. Because fibrillin-1 was known to be an . . . [Full Text of this Article]
