Gene therapy gives man with sickle cell disease the chance for a better future

For Evie Junior, living with sickle cell disease has been like running a marathon.“But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said. “It gets harder as you get older. Things start to fail and all you can think about is how much worse it’s going to get down the road.”In sickle cell disease, a genetic mutation causes the blood-forming stem cells — which give rise to all blood and immune cells — to produce hard, sickle-shaped red blood cells. These misshapen cells die early, leaving an insufficient number of red blood cells to carry oxygen throughout the body. Because of their sickle shape, these cells also get stuck in blood vessels, b locking blood flow and resulting in excruciating bouts of pain that come on with no warning and can leave patients hospitalized for days.The disease affects 100,000 people in the United States and millions around the world, the majority of whom are of African or Hispanic descent. It can ultimately  lead to strokes, organ damage and early death.As a child growing up in the Bronx, New York, Junior had to have his gall bladder and spleen removed due to complications from the disease, but he refused to let his condition limit him. He played football, basketball and baseball during the day, even though on some nights he experienced pain crises so severe he couldn ’t walk.“It was just really routine if I had a sickle cell crisis,” he said. “Going to the eme...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news