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Wasting
Muscles
Scientists edited dogs’ genes
to correct a common form
of muscle dystrophyDuchenne muscular dystrophy is a life-
threatening muscle-wasting illness. Occur-
ring mostly in males, it is the most com-
mon type of muscular dystrophy, striking
about one in 3,500 boys and causing their
muscles to start breaking down in early
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wheelchairs by the time they are teenagers
and usually leads to an early death from
heart or respiratory failure. There is no
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The disease is caused by gene muta-
tions 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 muscle protein levels in
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advance may hasten clinical trials for simi-
lar treatments in humans.
The research team, led by the University
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worked with young beagles bred to have
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muscle cells to remove a key barrier to high-
er protein production—a short, problematic
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in both canines and humans with the illness.
Within about two months the dogs were