Gene Therapy for Genetic Blood Disorders

Gene therapy was used to turn on the fetal hemoglobin (HbF) gene in HUDEP-2 cells (adult human red blood cell precursor), which is a model of the cells that form red blood cells in humans. HbF gene is usually turned off after birth resulting in low fetal hemoglobin levels. However, some individuals have increased HbF levels even in adulthood, a condition called Persistence of Fetal Hemoglobin (HPFH). Further, increased HbF levels in individuals suffering from genetic blood disorders result in milder symptoms. Introducing the HPFH responsible mutation in sickle cell anemic and thalassemia patients may ultimately lead to a cure for these hemoglobinopathies.