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News Link • Science, Medicine and Technology

New gene editing method corrects muscular dystrophy in mice

•, By Richard Moss
The technique, called CRISPR/Cas9-mediated genome editing, removes the mutation entirely in mice, and could have far-reaching consequences in the treatment of muscular dystrophy in people.

According to the Centers for Disease Control, DMD appears in approximately one out of every 3,500 male births in the US (but rarely appears in girls). It usually strikes before the age of six, often confining patients to a wheelchair before adolescence, with death generally before age 25.

It is a severe form of muscular dystrophy caused by a mutation in a gene called dystrophin that leads to loss of function and strength, not only in voluntary muscles such as those in the arms and legs, but also (later) in the cardiovascular system. It has no cure and existing treatments focus on improving quality of life more so than on halting the progression of the disease.

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