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

| | February 27, 2019
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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.

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.

Related article:  Bluebird Bio defends as 'simple and human' its pricing of costly gene therapy for beta thalassemia

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

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