How Does Gene Therapy for Sickle Cell Work? 5 Things You Need to Know

Sickle cell disease (SCD) is a genetic disorder affecting hemoglobin, the protein in red blood cells that carries oxygen. In SCD, the red blood cells become misshapen (sickle-shaped), leading to blockages in blood vessels, chronic pain, and organ damage. Until recently, the primary treatments for SCD were blood transfusions, pain management, and in rare cases, stem cell transplants. However, a new breakthrough in gene therapy offers hope for a lasting solution. What is Gene Therapy for Sickle Cell Disease? Gene therapy for sickle cell disease involves editing or replacing faulty genes responsible for the sickle-shaped blood cells. The primary goal of gene therapy is to correct the underlying genetic mutation, allowing the body to produce healthy red blood cells that do not sickle. Unlike traditional treatments that manage symptoms, gene therapy has the potential to offer a one-time cure by targeting the root cause of the disease. “Currently, the only cure for sickle cell disease is a stem cell transplant. It’s a potentially permanent solution, but it’s very challenging,” Yvette Miller, executive medical director of the American Red Cross, tells BlackDoctor.org. How Gene Therapy Works The gene therapy process for SCD is complex and can take up to two years...