The help won't come too soon for sickle cell patients, who suffer when their elongated or sickled cells try to squeeze through blood vessels designed for round cells. The disease causes organ damage because the deformed red blood cells cannot do their job of transporting oxygen. Sickle cell disease is a genetic disorder that afflicts about 60,000 African Americans. One in 500 African Americans has the disease, and one in 12 carries the genetic trait. Sickle cell anemia, the most severe form of sickle cell disease, affects about 2,000 Alabama residents and an estimated 148,000 carry the genetic trait, according to figures from Alabama's Sickle Cell Disease Association.
Some scientists propose certain gene therapies for the treatment of sickle cell disease.
Several different scientists have experimented with drug treatments to increase fetal hemoglobin, a type of hemoglobin that helps decrease the number of cells that sickle. But Goodman said he's the first to study blocking the formation of the irreversibly sickled cells with drug therapies. His team has found that red blood cells that become irreversibly deformed do so because of damage to a protein called beta-actin. When this damage occurs, a type of bridge forms, changing the shape of the actin protein and locking the cellular skeleton into a sickle shape. Some cells remain in this shape even after being treated with oxygen and even though their hemoglobin appears to have returned to normal.