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New CRISPR-based sickle cell treatment, explained

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STAT

STAT

8 ай бұрын

The Food and Drug Administration has approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder.
The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations - ushering in a new era of genetic medicines for inherited diseases.
This new video from STAT explains how the drug works, and how it is manufactured.
More STAT coverage on this treatment here: www.statnews.c...
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