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What happens when you CRISPR people?

Few questions generated more contentious discussion in biotech in the mid-2010s, as researchers and executives debated the relative merits of preclinical studies that pointed both to the new gene-editing tool’s potential to cure numerous diseases and its potential to cause unintended genetic damage.


Over the last three years, though, data have slowly emerged to provide a tantalizing answer: A CRISPR-based treatment has seemingly cured more than 70 clinical trial participants of sickle cell disease, a deadly and debilitating condition long ignored by the medical establishment, and a related disease called beta-thalassemia. Approval of the treatment, developed by Vertex, could come as soon as next year.

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