A one-time snip of a key gene lowered heart-clogging cholesterol by about 50% over an entire year with no adverse effects, scientists have reported.
The overwhelmingly positive results were found in four people who had taken the highest dose of the experimental treatment during a pilot study published in The New England Journal of Medicine in November 2025.
That study was extremely small — only 15 patients with dangerously high cholesterol that did not respond to medication.
The research was designed to test the safety of five different doses of a gene-altering infusion delivered by CRISPR-Cas9, a biological scissor which cuts a targeted genetic location to modify or turn a gene on or off. (CNN)
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