Sickle Cell Disease (SCD) is an inherited disorder that results in premature mortality and severe morbidity in millions of afflicted individuals worldwide. Novel therapies are urgently needed and research to improve therapies for SCD is an NHLBI priority. Clinical trials using new methods of gene manipulation have begun in adults; however, it is most desirable to treat children, adolescents and young adults (AYA) before the onset of irreversible organ damage.