U.S. patients with sickle cell disease now have a novel treatment option: the first-ever CRISPR-based therapy.
On December 8, the U.S. Food and Drug Administration approved the gene-editing therapy for use in patients age 12 years and older. In addition to offering hope of relief for people with severe forms of the painful blood disorder, the treatment, called Casgevy, is the world’s first to genetically tweak cells using the Nobel Prize–winning molecular scissors CRISPR/Cas9 (SN: 10/7/20).
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