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With the snip of a gene, scientists hope to erase high cholesterol for life
A 15-patient pilot study found the highest dose cut LDL nearly 50% and triglycerides 48% after one year, researchers said.
On Friday, an update published in The New England Journal of Medicine showed a one-time CRISPR-based gene-editing infusion significantly lowered LDL cholesterol and triglycerides for at least one year in a pilot study.
Researchers based the treatment on a genetic mutation in the ANGPTL3 gene that naturally regulates cholesterol, with the highest 0.8 milligram per kilogram dose showing the most durable reductions.
Participants receiving the highest dose saw average triglyceride decreases of 55% at two months, with cholesterol levels dropping by 53% at one year, while reported side effects remained minimal.
Lead study author Dr. Luke Laffin noted the reduction appears "durable and safe" in high-dose recipients, though the study remains small with only 15 patients and requires further Food and Drug Administration approval.
A new clinical trial with 40 participants is underway in the U.S. and Australia, with the FDA recommending 15 years of follow-up monitoring to identify potential long-term adverse effects.