![]() ![]() In this review, we will focus on the most important advances in the last decade. Other advances have contributed to the FDA approval of three new medications in 20 for management of sickle cell disease, with several other drugs currently under development. Alongside therapeutic reactivation of fetal hemoglobin, further understanding of stem cell transplantation and mixed chimerism as well as gene editing, and genomics have yielded very encouraging outcomes. In the last 10 years, discovery of BCL11A, a major γ-globin gene repressor, has led to a better understanding of the switch from fetal to adult hemoglobin and a resurgence of efforts on exploring pharmacological and genetic/genomic approaches for reactivating fetal hemoglobin as possible therapeutic options. Unfortunately, the translation of such knowledge into developing treatments has been disproportionately slow and elusive. Elucidation of its molecular basis prompted numerous biochemical and genetic studies that have contributed to a better understanding of its pathophysiology. Sickle cell anemia (SCA) was first described in the Western literature more than 100 years ago.
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