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Duchenne Muscular Dystrophy (DMD) Treated With Gene-Editing Technique

First Posted: Dec 31, 2015 09:24 PM EST

Duchene muscular dystrophy (DMD), an inherited, progressive form of muscular dystrophy that is more common in boys than girls, is estimated to occur in about 200,000 individuals each year in the United States. Though there are treatments that can ease symptoms related to the illness, there is no cure at this time.

Now, scientists at the University of Texas Southwestern, Harvard University and Duke University are using gene editing to correct a mutation that results in the disorder. Researchers used the CRISPR/Cas9 method of gene editing to correct a mutation that causes the health issue.

The health issue, which is linked to an x-linked DMD gene that encodes the protein dystrophin, was reversed when researches corrected the mutation in mice models. They did this by snipping away one of the genetic mutations thought to be responsible for the heath issue. Then, researchers found that the injected mice showed some restored muscle function. 

"We are very encouraged by these results in animals and feel this work is an important step forward in realizing the potential of gene editing to correct disease-causing mutations in muscle tissue of patients with DMD," said Amy Wagers, a study author at Harvard University.

"This study represents a very important translational application of genome editing of DMD mutations in young mice," said Dr. Rhonda Bassel-Duby, a professor of molecular biology at the Wellstone Center at UT Southwestern, in a news release. "It's a solid step toward a practical cure for DMD."

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