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Scientists stop Duchenne muscular dystrophy with gene editing

Progression of the disease was stopped by researchers using the CRISPR/Cas9 method of gene editing.
By Stephen Feller   |   Dec. 31, 2015 at 3:48 PM

DALLAS, Dec. 31 (UPI) -- Scientists used the CRISPR/Cas9 method of gene editing to correct a mutation causing Duchenne muscular dystrophy, reversing the progressive disease in the experiments with mice, according to a new study.

DMD is the most common, and severe, form of muscular dystrophy among boys. The progressive disease is caused by a mutation in the x-linked DMD gene that encodes the protein dystrophin.

The study conducted at the University of Texas Southwestern is one of three published in the journal Science -- the others were conducted at Harvard University and at Duke University.

All three studies found similar results using the groundbreaking gene editing method, which utilizes viruses to sneak proteins into cells to correct the mutated genes.

"This is different from other therapeutic approaches, because it eliminates the cause of the disease," said Dr. Eric Olson, chair of the department of molecular biology at the University of Texas Southwestern Medical Center, in a press release.

Researchers sent gene-editing components to cells in mice using the adeno-associated virus 9, and the rodents started producing dystrophin and showing improvement to skeletal muscle and the heart.

"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. "It's a solid step toward a practical cure for DMD."

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