UK approves gene therapy to cure two blooddisorders - BBC News

In a world first, medical regulators in the UK haveapproved a gene therapy that aims to cure sickle celldisease and beta thalassemia.

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

It is a revolutionary advance for two inherited bloodconditions, both triggered by errors in the gene forhaemoglobin.

In trials, 28 out of 29 sickle cell patients were free ofsevere pain and 39 of 42 beta thalassemia patients nolonger needed blood transfusions for at least a year.
The trials are continuing in the UK, US, France,Germany and Italy.

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