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CRISPR deployed to combat sickle-cell anaemia
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 Medicine, 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.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.