Super Hopes for Supercells

US researchers reported promising early results treating sickle cell disease using CRISPR technology this week.
Edited “super cells” in a patient appear to be responding as hoped—mass producing a critical protein lacking in most patients with the debilitating disease. Still, long-term success is unknown.

Vertex Pharmaceuticals and CRISPR Therapeutics—the study’s 2 sponsor companies—also saw early success treating a German patient with beta thalassemia, a similar disorder. 

“This is the first evidence that the new CRISPR technology has the potential to be curative for serious genetic diseases,” says Vertex’s David Altshuler. 


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