Scientists have reached a major milestone by editing a gene that could permanently lower dangerously high cholesterol. This could mean people might not need medicine for life. In a small pilot study with 15 patients, CRISPR-Cas9 technology targeted the ANGPTL3 gene, which controls LDL (bad cholesterol) and triglycerides. The study was published in the New England Journal of Medicine.
Results showed nearly a 50% drop in LDL cholesterol and a 55% decrease in triglycerides at the highest dose, marking a big advance in heart health management. Experts believe this one-time gene therapy could be a long-term solution, especially for younger people with severe cholesterol issues.
Heart disease is the leading cause of death worldwide. Current treatments need daily medication, which is hard for many to manage. This gene-editing mimics a rare natural mutation that turns off the ANGPTL3 gene, protecting the heart without harm.
Researchers advise continuing medications until more tests prove long-term safety and effectiveness. Phase 2 and Phase 3 trials are expected by the end of next year to expand this treatment to millions globally.
This breakthrough offers hope for a future with a single gene therapy drastically reducing heart disease risk.
