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People with the genetic disease Duchenne muscular dystrophy cannot synthesize dystrophin, a component of the scaffolding inside muscle cells. This deficiency results in muscle wasting and early death. The CRISPR–Cas9 gene-editing system has previously been used to trigger dystrophin production in mice. A team of scientists at the University of Texas Southwestern Medical Center in Dallas sought to test the technique in dogs. The researchers treated four dogs — all lacking dystrophin because of a genetic mutation — with the CRISPR–Cas9 system, which snipped out a short stretch of the animals’ DNA. This allowed the dogs’ cells to make dystrophin. Levels of the protein in one dog’s heart muscle reached 92% of normal.

 

Source: Nature, 31 August 2018.

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