Tuesday, September 10, 2024

Gene-editing on the NHS to transform lives of blood patients

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Since she was a baby, Kirthana Balachandran has needed a blood transfusion every three weeks to keep her alive.

Now 21, she has had hundreds of transfusions which have left her veins “bruised and scarred” to cope with the rare blood disorder transfusion-dependent beta thalassemia (TDT).

But Balachandran could now look forward to a future free of cannulas and needles, because a world-first gene-editing therapy is becoming available on the NHS, in a “historic moment” for the health service.

The treatment, called Casgevy, is the first to be licensed using the Nobel-prize-winning gene-editing tool Crispr. NHS patients will be among the first in the world to be treated with the therapy, after the National Institute for Health and Care Excellence (Nice) approved it for

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