In a world first, the UK medicines regulator has approved a therapy that uses the CRISPR–Cas9 gene-editing tool as a treatment. The decision marks another high point for a biotechnology that has been lauded as revolutionary in the decade since its discovery.
The therapy, called Casgevy, will treat the blood conditions sickle-cell disease and β-thalassaemia. Sickle-cell disease, also known as sickle-cell anaemia, can cause debilitating pain, and people with β-thalassaemia often require regular blood transfusions.
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