animal experiments

posted on July 1, 2005 - 2:53pm
QUEST Vol. 12, No. 4
In 1984, two years before mutations in the X-chromosome gene for the muscle protein now known as dystrophin were identified as the cause of Duchenne MD, researchers at the University of California at Berkeley and an agricultural center in Scotland announced they’d found an X-linked muscular dystrophy in mice. (Doctors had long known that DMD was a genetic disease and that, because of its...

What can laboratory studies tell us about human disease?

posted on July 1, 2005 - 2:39pm
QUEST Vol. 12, No. 4
This spring, the Duchenne muscular dystrophy community rejoiced when PTC Therapeutics of South Plainfield, N.J., announced that an experimental drug aimed at the disease had passed safety tests in human volunteers and would now be tested in boys with DMD. This drug, like many others, was successfully tested in mice lacking dystrophin, the protein that’s also missing in the human form of the...