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Studies test whether gene-editing can fix high cholesterol. For now, take your medicine

In early trials, a one-time liver infusion of CRISPR switched off ANGPTL3 genes, and 15 adults in the high-dose group saw LDL and triglycerides fall by half within two weeks.
Years ago, genetic studies found loss‑of‑function mutations in ANGPTL3 and PCSK9 naturally produce very low LDL and low heart‑disease risk, while millions on statins often struggle with lifelong therapy and want a durable, one‑time solution, Dr. Luke Laffin said.
Initial trials, run in Australia and the U.K., indicate major safety unknowns like off-target editing and liver irritation, while gene edits in mice have lasted a lifetime and companies pursue multiple gene targets.
A Lilly spokesperson said U.S. study sites are opening and CRISPR Therapeutics' next-step trial should start later this year, while some Verve Therapeutics participants have been tracked for two years with cholesterol still lowered.
Cardiovascular disease remains the leading killer, and the American Heart Association lists eight key factors, urging diet, exercise, and blood pressure target less than 120 over 80 with LDL targets: 100 for healthy people and at least 70 for those at high risk.

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Studies Test Whether Gene-Editing Can Fix High Cholesterol

(MedPage Today) -- Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol. It's very early stage research, tried in only a few dozen people so far. But gene...

1 day ago·New York, United States

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