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CRISPR Stops Rare Genetic Disease In Groundbreaking Human Trial

Gene-editing technique CRISPR may deliver new treatments for genetic diseases—and it’s already being tested on patients.


17:22 minutes.

In one of the first clinical applications of the technique, last month researchers reported in the New England Journal of Medicine that CRISPR had stopped a genetic disease called amyloidosis, which occurs when an abnormal protein accumulates in your organs. They’re not the only group moving toward using CRISPR on humans; recently, the FDA approved a human clinical trial that will use the technique to edit genes responsible for sickle cell disease.

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