For the first time, doctors within the U.S. have used the powerful gene-editing method CRISPR to attempt to treat a patient with a genetic disorder.
“It’s just wonderful how far issues have come,” says Victoria Gray, 34, of Forest, Miss. “It’s great,” she informed in an interview after undergoing the landmark remedy for sickle cell disease.
Victoria Gray is the first patient ever to be publicly recognized as being involved in a study testing using CRISPR for a genetic disease.
“I at all times had hoped that something would come along,” she tells from a hospital mattress at the Sarah Cannon Research Institute in Nashville, Tenn., where she acquired an infusion of billions of genetically modified cells. “It is a good time to get healed.”
But it most likely will take months, if not years, of careful monitoring of Gray and different patients before doctors know whether or not the therapy is safe and how effectively it might be serving to sufferers.
Sickle cell affects thousands of individuals around the world. About 100,000 are within the U.S., and most of them, like Gray, are African American. A genetic defect causes bone marrow to provide a defective protein that makes blood cells, which are sickle-shaped, sticky, and hard. The disfigured cells get stuck inside blood vessels and do not carry oxygen usually, causing a host of debilitating and, often, eventually life-shortening issues.
“It is horrible,” Gray says. “When you can’t walk or lift a spoon to feed yourself, it will get actual hard.”