‘First ever’ human CRISPR trial targets rare beta thalassemia blood disorder

CRISPR, the groundbreaking and controversial gene-editing technique, has been used in a patient with a debilitating blood disease, scientists revealed [February 25].

The US and Swiss companies behind the venture claim to be the first ever to use CRISPR on a human, disregarding the use of it on cancer patients in China.

The unidentified patient in the clinical trial has beta thalassemia, a hereditary condition that turns off a crucial gene, hampering their ability to make hemoglobin, which is needed to push oxygen around the body.

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But Swiss biotech company CRISPR Therapeutics, backed by Boston’s Vertex Pharmaceuticals, has attempted to cure the disease using the experimental DNA-editing technique CRISPR to switch the defective gene back on.

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The firms say they are soon to do the same with another patient who has sickle cell anemia, a blood condition that causes excruciating pain.

The scientists hope the treatment – one injection of a harmless virus to snip out and delete the defect, followed by a stem cell injection to insert a ‘healthy’ copy of the gene – will be a one-and-done cure.

Read full, original post: First human in gene-editing trial infused with CRISPR to ‘cure’ rare blood disorder