A Genetic Cure for Sickle Cell Disease Moves Closer to Reality
Gene therapy and CRISPR show promising signs
Millions of people around the world, including around 100,000 in the United States, suffer from sickle cell disease, a brutally painful inherited blood disorder. Most of them are of African descent. Over time, the disease worsens and can cause infections, organ damage, blindness, stroke, and early death.
“I can’t think of a more miserable disease than sickle cell,” James Taylor, director of the Howard University Center for Sickle Cell Disease in Washington, D.C., tells OneZero.
In the United States, sickle cell patients have long endured poor care and discrimination because of deep-rooted inequities in health care. A cure for the disease currently exists but is out of reach for many patients. A recent boom in sickle cell research, however, could make a cure available to many more people. New results from two experimental genetic treatments show promise for eliminating the disease.
Sickle cell arises from a mutation in a gene that makes a critical blood protein called hemoglobin. People who inherit this mutation from both parents have an abnormal form of hemoglobin that produces sickle-shaped red blood cells instead of healthy round ones. These sickled blood cells clump together in the bloodstream and cause painful episodes lasting hours or days. People with sickle cell frequently end up in the hospital because of these bouts of pain, and many need regular blood transfusions to provide normal blood cells to the body.
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“These patients could die during any one of those hospitalizations, and they live with that sword hanging over their head,” Samarth Kulkarni, CEO of CRISPR Therapeutics, tells OneZero. “After having our treatment there have been no hospitalizations to date.”
In a pioneering clinical trial run by CRISPR Therapeutics and Vertex Pharmaceuticals, a single infusion of genetically modified cells…