“Treatment with curative intent”: the emergence of genetic therapies for sickle cell anemia

Abstract

Abstract The root cause of sickle cell anemia has been known for 7 decades, yet no curative therapies have been available other than allogeneic bone marrow transplantation, for which applicability is limited. Two potentially curative therapies based on gene therapy and gene editing strategies have recently received US Food and Drug Administration approval. This review surveys the nature of these therapies and the opportunities and issues raised by the prospect of definitive genetically based therapies being available in clinical practice.