Oncohema Key

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…

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…

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…

There are multiple effective and well-tolerated systemic therapy treatments for the treatment of advanced melanoma, as well as new immunotherapy and targeted therapy agents in clinical trials. Traditional cytotoxic chemotherapy…