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Landmark CRISPR Trial Shows Promise Against Deadly Disease
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Landmark CRISPR Trial Shows Promise Against Deadly Disease

Administering gene-editing treatment directly into the body could be a safe and effective way to treat a rare, life-threatening condition.

Heidi Ledford
Nature, 2021
References: Gillmore et al. (2021)
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Previous gene-editing therapies involved removing cells from the body, revising their genetic material, then re-infusing the cells. A breakthrough clinical trial now proves that gene-editing components called CRISPR-Cas9 can be injected directly into the body with astonishing results, offering hope to those suffering from at least one painful, life-threatening disease. Scientists think similar therapies could improve the lives of countless others.

Summary

Early clinical trial data suggest that CRISPR-Cas9 gene editing can successfully treat life-threatening disease.

A groundbreaking study shows that the infusion of a gene-editing substance that attacks an errant protein made by the liver can prove effective and safe.

In a clinical trial, six subjects afflicted with a rare, fatal medical condition called “transthyretin amyloidosis” received one treatment of CRISPR-Cas9 gene-editing components. All patients saw a reduction in a “misshapen protein” called TTR, which is endemic to the disease. Higher doses of CRISPR-Cas9 related to an approximate 87% decline in irregular protein levels.

Intellia Therapeutics of Cambridge, Massachusetts, working with Regeneron of Tarrytown, New York, developed the treatment and published results in The New England Journal of Medicine. Other CRISPR-Cas9 trials have used a technique where cells are removed from the body, edited and then re-infused. Editing genes within the body opens...

About the Author

Heidi Ledford is a senior reporter for Nature. She specializes in biomedical topics such as cancer research, drug development, biotechnology and CRISPR.

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