Sickle cell disease: a new era

Sickle cell disease is caused by inherited mutations of the globin gene, and is a multisystem disorder characterised by distortion, stiffness, and adhesion of red blood cells. Every year in Africa, around 230 000 children are born with sickle cell disease and about 90% of them could die before the age of 5. At around 1 year of age, these children begin to have anaemia, pain, stroke, retinopathy, and chronic damage affecting the spleen, lungs, kidney, and major joints. They become particularly susceptible to infection—pneumococcal sepsis is the leading cause of death.