CRISPR gene therapy seems to cure dangerous inflammatory condition

Key Points:

  • Nine people with hereditary angioedema appear to have been cured with a new CRISPR-based gene therapy
  • The therapy involves making cuts in the gene for kallikrein, a protein involved in inflammation
  • The treatment was given directly into the body, with participants experiencing a 95% reduction in swelling attacks


A groundbreaking new approach shows promise in treating a rare genetic condition known as hereditary angioedema. This innovative therapy, utilizing CRISPR technology, targets the gene responsible for kallikrein, a protein linked to inflammation.


In a trial involving nine individuals afflicted with this debilitating condition, results have been nothing short of astounding. Participants experienced a significant reduction in swelling attacks following the treatment, with some reporting no further episodes for over a year. This remarkable outcome has raised hopes of a potential cure for hereditary angioedema.


Unlike traditional therapies that focus on managing symptoms, this gene therapy aims to address the root cause of the condition by directly targeting the gene responsible for producing kallikrein. Administered in a single infusion, this ‘in vivo’ delivery method offers a streamlined and efficient approach compared to the more complex ‘ex vivo’ procedures seen in other CRISPR-based therapies.


Researchers and experts in the field are optimistic about the broader implications of this approach, envisioning its potential application in treating a range of genetic disorders. With the recent approval of similar genetic therapies for conditions like sickle cell disease and beta-thalassaemia, the success of this trial paves the way for a new era in precision medicine.



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