In this article, the authors discuss new approaches to treating iron overload diseases using hepcidin mimetics or by modulating endogenous hepcidin expression. In particular, the authors discuss lipid nanoparticle encapsulated…
Use of new compound such as inhibitors of JAK2 or transforming growth factor β-like molecules might soon revolutionize the treatment of β-thalassemia and related disorders. However, this situation requires careful…
Recent studies suggest that sickle cell disease (SCD) is a hypercoagulable state contributing to vaso-occlusive events in the microcirculation, resulting in acute and chronic sickle cell–related organ damage. In this…
The pathophysiology of vasoocclusion is thought to involve a wide variety of adhesive interactions involving erythrocytes, leukocytes, and the endothelium. Selectins are expressed by leukocytes, platelets, and the endothelium, among…
P-selectin on endothelial cell surfaces is central to impaired microvascular blood flow in sickle cell disease (SCD). Restoration of blood flow is expected to provide therapeutic benefit for SCD patients,…
Low global arginine bioavailability (GAB) is associated with numerous complications of SCD including early mortality. Mechanisms of arginine dysregulation involve a complex paradigm of excess activity of the arginine-consuming enzyme…
Data suggest a role for adenosine signaling in the pathogenesis of sickle cell disease (SCD). Signaling through the adenosine A 2A receptor (A 2A R) has demonstrated beneficial effects. Activation…
Inflammation plays a critical role in the complex pathophysiology of sickle cell disease and drives both the acute and chronic processes leading to vascular injury. Mediators of inflammation, such as…
This review presents the indications and contraindications (pros and cons) for the potential use of erythropoietin (Epo) as a treatment in β-thalassemia and sickle cell anemia (SCA). Its high cost…
Fetal globin (gamma globin; HBG) is normally expressed during fetal life and prevents the clinical manifestations of beta hemoglobinopathies before birth. HBG genes are normally integrated in hematopoietic stem cells…
