Last year I posted four articles on CRISPR. The technology is new and fascinating. There appears to be endless possibilities as to what it might be able to do someday. For those of us living with a neuromuscular condition (including Kennedy’s Disease), it offers hope.
In this latest article written by Kate Lunau for McLean’s Magazine, researchers have taken another step forward in this quest for a treatment or cure.
Gene editing spurs hope for muscular dystrophy cure
For the first time, a breakthrough technique called CRISPR has been used to treat a genetic disease inside a living mammal
Kate Lunau January 4, 2016
“Of the many diseases that afflict us, those that strike young children seem especially cruel. One is Duchenne muscular dystrophy, which typically appears before a child’s sixth birthday. Sufferers (almost always boys) struggle with muscle weakness, and have trouble walking. By 12, most will be in a wheelchair; few live beyond their thirties. There’s no cure, and it affects about one in 3,500 males born worldwide. Suddenly, with the arrival of a powerful gene editing technique, new treatments seem to be on the horizon. In three independent papers, published in the journal Science, U.S. scientists snipped out defective DNA in mice with the disease—the first time this tool, called CRISPR, has been used to treat any genetic disease inside a living mammal—and restored some of their muscle function. ...”