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Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, according to a study published today in Blood Advances. SCD is an inherited red blood cell disorder affecting approximately 100,000 people in the United …
A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients. Four patients at Columbia University Irving Medical Center/New York-Presbyterian participated in the multicenter study, …
Overview Sickle cell disease is a group of inherited red blood cell disorders that affects hemoglobin, the protein that carries oxygen through the body. Normally, red blood cells are disc-shaped and flexible to move easily through the blood vessels. If you have sickle cell disease, your red blood cells are crescent or “sickle” shaped. These …
Researchers from the University of California, Irvine and the University of Minnesota have found that an enriched diet and companionship can reduce pain in mice with sickle cell disease by increasing serotonin. They also discovered that duloxetine, an antidepressant that boosts serotonin levels, could be an alternative to opioids in treating chronic pain. “Finding safe …
Sickle cell disease, a red blood cell disorder that causes cellular deformation resulting in cells similar in shape to sickles, is the most common inherited blood disorder in the United States and affects approximately 100,000 Americans. The vulnerable cells have a shorter lifespan than normal cells and thus, there is a decreased amount of oxygen …