Three groups reported in Science on Dec. 31st that they used CRISPR to selectively edit the defective gene responsible for Duchenne muscular dystrophy (DMD) in mice, allowing the animals to make an essential muscle protein. The approach is the first time CRISPR has been successfully delivered throughout the body to treat grown animals with a genetic disease.
DMD stems from defects in the gene coding for dystrophin, a protein that helps strengthen and protect muscle fibers. The rare disease affects approximately 1 in every 5,600 to 7,700 males ages 5 through 24 in the U.S. The researchers used harmless adeno-associated virus to carry the CRISPR-Cas9 gene editing system, which snips away the genetic mutations for DMD in living mice. In the treated mice, which had CRISPR-ferrying viruses injected directly into muscles or into their bloodstream, heart and skeletal muscle cells made a truncated form of dystrophin, and the rodents performed better on tests of muscle strength than untreated DMD mice.
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