With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for medication, according to a new pilot study published Saturday in the New England Journal of Medicine.
The study was extremely small — only 15 patients with severe disease — and was meant to test the safety of a new medication delivered by CRISPR-Cas9, a biological sort of scissor which cuts a targeted gene to modify or turn it on or off.
Preliminary results, however, showed nearly a 50% reduction in low-density lipoprotein, or LDL, the “bad” cholesterol which plays a major role in heart disease — the No.1 killer of adults in the United States and worldwide.
The study, which will be presented Saturday at the American Heart Association Scientific Sessions in New Orleans, also found an average 55% reduction in triglycerides, a different type of fat in the blood that is also linked to an increased risk of cardiovascular disease.
“We hope this is a permanent solution, where younger people with severe disease can undergo a ‘one and done’ gene therapy and
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