Breakthrough in CRISPR Gene Therapy Shows Promise for Heart Disease

In what researchers are calling a watershed moment for genetic medicine, a Phase 2 clinical trial has demonstrated that a single CRISPR-based gene editing treatment can permanently reduce LDL cholesterol levels by up to 55%. The trial, conducted across 12 medical centers in North America and Europe, involved 382 patients with familial hypercholesterolemia. The treatment works by precisely editing the PCSK9 gene in liver cells, effectively turning off the body's production of a protein that prevents cholesterol removal from the bloodstream.