UK approves gene therapy to cure two blood disorders – BBC News

In a world first, medical regulators in the UK have approved a gene therapy that aims to cure sickle cell disease and beta thalassemia.

The treatment is the first to be licensed using the gene-editing tool known as Crispr, for which its discoverers were awarded the Nobel prize in 2020.

It is a revolutionary advance for two inherited blood conditions, both triggered by errors in the gene for haemoglobin.

In trials, 28 out of 29 sickle cell patients were free of severe pain and 39 of 42 beta thalassemia patients no longer needed blood transfusions for at least a year.

The trials are continuing in the UK, US, France, Germany and Italy.

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