About the book
Beta-thalassemia is classified into three main subgroups based on their clinical expression: major, intermedia, and minor. β-thalassemia major presents itself within the first 2 years of life with severe anemia, poor growth, and skeletal abnormalities and requires regular, lifelong blood transfusions. Thanks to the significant improvement in therapy, patients with β-thalassemia may reach an advanced age. This is associated with clinical symptoms that are the consequence of the disease itself and the treatment modalities. Agents such as JAK2 inhibitors and TGF-β ligand traps that reduce the ineffective erythropoiesis process are already being tested in clinical trials with promising results. Other agents that aim to reduce oxidative stress (activators of Foxo3, HRI-eIF2aP, Prx2, Hsp70, and PK anti-oxidant systems and inhibitors of HO-1) and to decrease iron overload (hepcidin agonists, erythroferrone inhibitors and exogenous transferrin) are also under experimental investigation. Significant progress has also been made in the area of allogeneic hematopoietic stem cell transplantation with several ongoing clinical trials examining new condition regimens as well as different donor selection and stem cell source options. Gene therapy has reached a critical point and phase 1 clinical trials have recently been launched to examine the effectiveness and especially long term safety. Epigenetic manipulation and genomic editing of the γ- or β-globin gene are novel and promising experimental gene therapy approaches for β-thalassemia giving hope for cure for this chronic disease.
This book will shed the light on recent findings regarding the pathophysiology underlying the major symptoms of β-thalassemia and potential therapeutic modalities for the amelioration of its complications, as well as new modalities that may provide a cure for the disease, also we will outlines the key points of the molecular mechanisms underlying β-thalassemia in relation to the development of new therapies and an update is given both at the pre-clinical and clinical level.