One-time gene edit slashes bad cholesterol by half

A single infusion of a CRISPR-Cas9 gene-editing therapy lowered LDL cholesterol by 50% in a Cleveland Clinic Phase 1 trial. Fifteen patients with lipid disorders that do not respond to standard drugs received the treatment. Within two weeks, LDL fell by half and triglycerides dropped about 55%, and the reductions persisted for at least 60 days. No serious treatment-related adverse events were reported during short-term follow-up. The findings were presented at the American Heart Association's Scientific Sessions 2025 and appeared in the New England Journal of Medicine.
Patients with stubborn high cholesterol now take a pill every day for life. One day, a single treatment might be enough.
Source: Cleveland Clinic First-In-Human Trial of CRISPR Gene-Editing Therapy Shown to Safely Lower Cholesterol and Triglycerides (newsroom.clevelandclinic.org).
Written by the HappyGood desk from the primary source linked above and checked against it. Every story links its source. Corrections: [email protected].
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