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Informationen zum Autor Elliott P. Vichinsky is the editor of Cooley's Anemia: Ninth Symposium, Volume 1202, published by Wiley. Ellis Neufeld is the editor of Cooley's Anemia: Ninth Symposium, Volume 1202, published by Wiley. Klappentext Thanks to scientific advances, individuals with thalassemia--a group of genetic blood disorders which includes Cooley's Anemia -- are now living into their 40's and 50's. Not only are individuals living longer, but their quality of life has increased. Scientific and clinical advancements have resulted in new iron-chelating drugs, early detection of organ failure, an understanding of adult complications associated with living with thalassemia (osteoporosis, heart failure, growth hormone deficiency, pulmonary hypertension, and infertility) and promising progress towards the ultimate magic bullet--a cure in the form of bone marrow and cord blood transplants, or gene therapy.This volume integrates basic science and clinical research so that both scientists and clinicians can develop a mutual understanding of recent progress in thalassemia.NOTE: Annals volumes are available for sale as individual books or as a journal. For information on institutional journal subscriptions, please visit www.blackwellpublishing.com/nyas. Zusammenfassung * Explores recent scientific and clinical advances in the treatment of thalassemia * Examines research working to build an understanding of adult complications associated with living with thalassemia * Looks at research making progress toward finding a cure through bone marrow and cord blood transplants, or gene therapy. Inhaltsverzeichnis Clinical trials in thalassemia: insights from the patient community Pathogenesis and management of iron toxicity in thalassemia The role of antioxidants and iron chelators in the treatment of oxidative stress in thalassemia Thalassemia as a global health problem: recent progress toward its control in the developing countries Iron metabolism and ineffective erythropoiesis in [beta]-thalassemia mouse models Hepcidin in [beta]-thalassemia Therapy for [beta]-globinopathies: a brief review and determinants for successful and safe correction Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in [beta]-thalassemia Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease Transcriptional silencing of fetal hemoglobin by BCL11A The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in [beta]-thalassemia patients Deferiprone Combined iron chelation therapy Deferasirox: current knowledge and future challenges Monitoring the efficiency of iron chelation therapy: the potential of nontransferrin-bound iron Critical appraisal of growth retardation and pubertal disturbances in thalassemia Magnetic resonance assessment of iron overload by separate measurement of tissue ferritin and hemosiderin iron Predicting pituitary iron and endocrine dysfunction Better survival and less cardiac morbidity in female patients with thalassemia major: a review of the literature. A preclinical approach for gene therapy of [beta]-thalassemia The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia Emerging insights in the management of hemoglobin E beta thalassemia Fetal globin gene inducers: novel agents and new potential Detection of the cardiovascular complications of thalassemia by echocardiography Cardiovascular MRI in thalassemia major Com...
