Muscular Dystrophy Gene Riddle Cracked

From Bloomberg - A genetic flaw behind a common form of muscular dystrophy may be explained in research that suggests why some people with the variant gene are afflicted with the muscle-wasting disease. While scientists have known for more than a decade that facioscapulohumeral muscular dystrophy, or FSHD, is linked to genetic abnormalities on the end of chromosome 4, not everyone with these changes gets the disease, said Daniel Miller, a genetics researcher at the University of Washington in Seattle. The findings show that strings of genetic code surrounding a critical gene can either disable it or allow it to be expressed and damage cells, Miller said. The results may help scientists develop new drugs to treat the condition, which generally strikes children and causes weakness that begins in their facial muscles and shoulders, then spreads down the body, Miller said. About 1 in 20,000 people worldwide have FSHD, according to the report published today in the journal Science. “Prior to this, if you wanted to develop a drug, you didn’t know exactly which genes caused it,” Miller said. “This allows us to focus drug development on a specific biological target.” Read more here:

Muscular Dystrophy Gene Riddle Cracked