CRISPR Gene Editing

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.

“This is a landmark approval which opens the door for further applications of CRISPR therapies in the future for the potential cure of many genetic diseases,” said Kay Davies, a geneticist at the University of Oxford, UK, in comments to the UK Science Media Centre (SMC).   READ MORE...