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). Spotted via Science Daily.
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