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A mutation in a single DNA letter causes a painful and debilitating disease known as sickle-cell anaemia. Researchers have wrestled with this illness for more than 65 years, and have now added CRISPR–Cas9 gene editing to their armoury.
In a paper published 12 October in Science Translational Medicine1, researchers reported some success in correcting the mutation in mice, though they concede that human applications are still years away. The efficiency of the process is also slightly too low for practical use, cautions author Jacob Corn, a biochemist at the University of California, Berkeley.
But the advance — and other recent efforts — has given Corn hope that the first treatment to address the cause of sickle-cell anaemia could be only a few years away. “We now finally may have some paths to address the cause of the disease rather than the symptoms,” he says.
About 250,000 children around the world are born with sickle-cell anaemia each year. It is caused by a mutation that affects the shape of the oxygen-carrying protein haemoglobin in red blood cells. The result...