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Landmark CRISPR Trial Shows Promise Against Deadly Disease
Article

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.

Nature, 2021


Editorial Rating

8

Qualities

  • Analytical
  • Scientific
  • Concrete Examples

Recommendation

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.

Take-Aways

  • Early clinical trial data suggest that CRISPR-Cas9 gene editing can successfully treat life-threatening disease.
  • Previous to this experimental therapy, transthyretin amyloidosis was virtually untreatable.
  • While regarded as revolutionary, the new CRISPR-Cas9 therapy presents limitations.

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.