Approximately 100 000 Americans, primarily African Americans, have sickle cell disease.1 Worldwide, the burden of this disease is much greater with an estimated 300 000 affected infants born.2 A single nucleotide substitution causes the autosomal recessive disease: valine replaces glutamic acid in the sixth position of the β-globin protein. Despite the discovery of the etiology of sickle cell disease more than 60 years ago,3 little progress has been achieved in the treatment or cure of this chronic disease.
King AA, DiPersio JF. Reconsideration of Age as a Contraindication for Curative Therapy of Sickle Cell Disease. JAMA. 2014;312(1):33-34. doi:10.1001/jama.2014.7193