Nov. 15, 2007 -- Scientists today reported that they have reversed symptoms of myotonic dystrophy -- adults' most common type of musculardystrophy -- in lab tests on mice.
"This work should provide hope and encouragement to people with myotonicdystrophy and their families," University of Rochester neurologist CharlesThornton, MD, says in a news release.
Muscular dystrophies include more than 30 genetic diseases that weaken andbreak down skeletal muscles that control movement.
Thornton's team created a compound called a morpholino that serves as apatch to make up for a genetic glitch linked to myotonic dystrophy.
The scientists injected the morpholino into the leg muscle of mice withmyotonic dystrophy. A single shot of the morpholino reversed symptoms for up toeight weeks, the study shows.
The morpholino hasn't been tested in people.
But the tests in mice prove the notion that "a fundamental aspect ofthis genetic disease can be reversed even after it is very wellestablished," says Thornton.
"As we move forward, we should not be content to keep this conditionfrom getting worse. We should set our sights on making it better," hesays.
The findings appear in today's online edition of The Journal of ClinicalInvestigation.