Protein May Offer New Treatment for Muscular Dystrophy Sufferers

A naturally occurring protein that can take the place of the missing protein in people with Duchenne and Becker muscular dystrophy may offer an important new pathway for treatment, according to researchers. The protein, called utrophin, is 80% identical to the missing protein, but serves a different function in the cell and is normally present only in low quantities. Geneticist Kay Davies and her colleagues at Oxford University in England report in the Nov. 28 issue of the journal Nature that dystrophic mice genetically engineered to produce above-normal quantities of utrophin suffer much less muscle wasting than those that have not been altered.

The Muscular Dystrophy Assn. finds the research so promising that it is funding a program to identify drugs that will “turn on” the utrophin gene so that more is produced in cells throughout the body.