It’s on: the 1980s vs. the 2000s, brute force vs. precision, traditional gene transfer vs. newfangled gene editing. The contest is being waged on numerous fronts, but nowhere more intensely than over Duchenne muscular dystrophy: Which technique — gene transfer or gene editing — holds out the best hope for a true cure?
Several biotech companies are trying to develop genetic treatments for Duchenne, and although Cambridge, Mass.-based biotech Sarepta scored points for traditional gene transfer with encouraging results two months ago, the new kid on the block is nipping at its heels: scientists on Thursday reported using CRISPR-Cas9 gene editing to cure a canine version of Duchenne in four beagles, a significant step up from the usual lab mice.
It is the first published account of delivering CRISPR systemically (that is, throughout the body) in a large mammal.
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