The pathophysiology of sickle cell disease involves the polymerization of sickle hemoglobin in its T state, which develops under low oxygen saturation. One therapeutic strategy is to develop pharmacologic agents…
After nearly two decades of struggle, gene therapy for hemoglobinopathies using vectors carrying β or γ-globin gene has finally reached the clinical doorsteps. This was made possible by advances made…
Ischemia-reperfusion (I/R) physiology, also called reperfusion injury, instigates vascular and tissue injury in human disease states. This review describes why sickle cell anemia should be conceptualized in this fashion and…
Different pathways lead from the simple point mutation in hemoglobin to the membrane changes that characterize the altered interaction of the sickle red blood cell with its environment, including endothelial…
I would like to dedicate this book to Sir David Weatherall. David is the best scientist and clinician-teacher I’ve ever met. He makes the clinician want to be a scientist,…
Elliott P. Vichinsky, MD, Editor Hemoglobinopathies are a worldwide public health problem. Millions of people are affected, with over 400,000 annual births of new cases. Major, recent advances in understanding…
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…
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…
