
                         Roanoke Times
                 Copyright (c) 1995, Landmark Communications, Inc.

DATE: THURSDAY, August 19, 1993                   TAG: 9308190024
SECTION: NATIONAL/INTERNATIONAL                    PAGE: A-12   EDITION: METRO 
SOURCE: Associated Press
DATELINE: NEW YORK                                LENGTH: Short

GENE TREATMENT BOOSTS MUSCLES

Scientists have prevented a condition resembling muscular dystrophy in mice by using a gene treatment, a step that boosts the prospects of using gene therapy to treat human patients, researchers say.

The work shows that an inserted gene can make muscle work normally and that even an overactive gene will cause no harm, researchers said.

But the study doesn't answer other major questions about gene therapy for Duchenne muscular dystrophy, the most common childhood muscular dystrophy.

The disease strikes about once in every 3,500 male births. It weakens and destroys muscles, usually beginning around ages 3 or 4. Few patients survive beyond their mid-20s.

Duchenne arises from defects in a gene that is supposed to tell muscle cells how to make a protein called dystrophin.

The mouse work is presented in today's issue of the journal Nature by Jeffrey Chamberlain, an assistant professor of human genetics at the University of Michigan Medical School in Ann Arbor, with scientists there and at the universities of Iowa and Washington.

They worked with a strain of mouse that lacks a working dystrophin gene. The mouse doesn't develop Duchenne as a human would, but it does show a severe Duchenne-like wasting of a diaphragm muscle and abnormalities in other muscles. Researchers injected a miniature version of a working dystrophin gene into fertilized mouse eggs. Later, they found that mice that had taken up the gene had completely normal diaphragm muscles and other muscles.

Tests showed the diaphragm muscle worked normally, said Chamberlain, who noted that weakness in respiratory muscles is often lethal to Duchenne patients.

by CNB