Science / Medicine : Dystrophy Treatment Works in Mice Tests

An experimental treatment for the most severe form of muscular dystrophy has worked in two new studies in mice, and tests in human patients may begin this summer, scientists said. The mice belonged to a strain that lacks a protein called dystrophin in their muscles. Researchers discovered last year that the lack or abnormal production of the protein causes Duchenne muscular dystrophy, the most common and severe form of dystrophy in humans.

Duchenne muscular dystrophy, which causes a progressive weakening and wasting of voluntary muscles, is a genetic disorder that strikes about one in every 3,500 male babies in the United States.

The mouse experiments involved immature muscle cells called myoblasts, which normally help repair muscle fibers that have broken because of injury or strenuous exercise. Researchers injected healthy myoblasts into mice that lacked dystrophin and found that the myoblasts fused with muscle fibers and allowed them to begin producing dystrophin.