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Promising treatment for Duchenne muscular dystrophy developed with CRISPR gene editing

| | October 25, 2018

Duchenne muscular dystrophy is a life-threatening muscle-wasting illness. Occurring mostly in males, it is the most common type of muscular dystrophy, striking about one in 3,500 boys and causing their muscles to start breaking down in early childhood. It often confines patients to wheelchairs by the time they are teenagers and usually leads to an early death from heart or respiratory failure. There is no cure—but a genetic fix tested in dogs may offer new hope.

The disease is caused by gene mutations that make patients’ muscle cells unable to produce enough dystrophin, a protein that helps muscles absorb shocks and protects them against degradation over time. In a recent study, scientists used a gene-editing technique called CRISPR/Cas9 to pump up [dogs’] muscle protein levels.

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[The team] worked with young beagles bred to have Duchenne. The scientists edited the dogs’ muscle cells to remove a key barrier to higher protein production—a short, problematic segment of protein-coding DNA that occurs in both canines and humans with the illness. Within about two months the dogs were producing greater amounts of dystrophin; levels in skeletal muscle ranged up to 90 percent of normal.

Exactly how long one injection with CRISPR gene-editing machinery might last in human Duchenne patients remains unknown. [researcher Eric] Olson and his colleagues hope the intervention might be durable enough with a single dose.

Read full, original post: CRISPR Gene Editing Shows Promise for Treating a Fatal Muscle Disease

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