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CRISPR gene-editing therapy safely and continuously lowers cholesterol and triglycerides, first-in-human trial shows
A Cleveland Clinic first-in-human clinical trial has shown that a one-time infusion of a gene-editing therapy using CRISPR-Cas9 was effective and safe in reducing LDL ("bad") cholesterol and triglycerides in people with medication-resist…
Summary
The Phase I trial, which included 15 patients, aimed to see if the reductions in LDL cholesterol and triglycerides previously reported after two months would persist for 12 months. After 12 months, patients treated with the highest dose saw a 52.5% reduction from baseline in LDL cholesterol and a 47.8% reduction in triglycerides.
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