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Luke Laffin reports CRISPR gene-editing therapy provides durable cholesterol reduction for at least one year

Luke Laffin, a preventive cardiologist at the Cleveland Clinic, reported that a single-course CRISPR-Cas9 gene-editing treatment successfully lowered cholesterol levels for at least one year. The study, published in The New England Journal of Medicine, focused on the ANGPTL3 gene, which regulates lipids in the liver. Patients receiving the highest dose of the therapy saw a mean reduction in low-density lipoprotein (LDL) cholesterol of 53% and a mean reduction in triglycerides of 48% after one year. This one-time treatment mimics a naturally occurring genetic mutation that protects against heart disease. Researchers noted that the therapy was well-tolerated with minimal side effects, potentially offering a permanent alternative to daily medications like statins. The results suggest that the edited liver cells continue to produce new generations of cells with the mutation, ensuring long-lasting biological effects. CRISPR Therapeutics is currently advancing the program into Phase 1b clinical trials to further refine the results for patients with severe hypertriglyceridemia.

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