Scientists have tried for several years to restore strength to the muscles of patients with muscular dystrophy by inserting genes that make dystrophin, a crucial muscle protein. But their attempts have proved ineffecient and impermanent.
Now, scientists at Oxford University have tried another tack in animals that they say may have greater chances of success. Muscle cells make not only dystrophin but a similar protein called utrophin. In a letter published today in the journal Nature, Kay E. Davies and her colleagues report that fitting mice with extra utrophin genes markedly reduced the degeneration in their muscles. The mice were a special breed that has a muscular-dystrophy-like abnormality.
The hope, wrote Davies, is to find an oral drug for MD patients that would cause their utrophin genes to make extra utrophin, compensating for the deficiency of dystrophin.
“An advantage of this approach is that it does not require gene replacement, because the patients already have a functional utrophin gene,” commented scientists from the University of Iowa in an article accompanying today’s report.