Nov 14, 2023 - News

Gene-editing treatment cuts cholesterol in small study

Illustration of a magnifying glass showing the pixelated piece of a double helix.

Illustration: Maura Losch/Axios

A study led by Boston-based Verve Therapeutics showed for the first time that gene-editing can cut high cholesterol.

Why it matters: The experimental treatment could offer an answer for tackling heart disease, the nation's leading cause of death.

Driving the news: Verve presented the findings Sunday at an American Heart Association meeting in Philadelphia, showing promising results for patients with a genetic disorder that causes high cholesterol, the New York Times reported.

Reality check: The trial involved only 10 patients in the UK and New Zealand.

  • Only three patients received a high enough dose to result in a substantial drop in cholesterol for several months.
  • The treatment needs to be tested on more people and for a longer period of time to confirm the approach is effective and safe, per NPR.

Yes, but: The study, which is ongoing, is a win for CRISPR, the form of gene-editing used in this treatment.

  • "We're super excited. This is the first-ever evidence that one can actually rewrite a single DNA letter in the human liver and have a clinical effect. So we're thrilled," Dr. Sekar Kathiresan, Verve's executive officer, told NPR in an interview.

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