| Preface |
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Thalassemia: The Continued Challenge |
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1 | (10) |
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Keynote Address: The Challenge of Thalassemia for the Developing Countries |
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11 | (7) |
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Part I. The Natural History of Thalassemia Worldwide |
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Changing Patterns of Thalassemia Worldwide |
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18 | (7) |
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α-Thalassemia: Hb H Disease and Hb Barts Hydrops Fetalis |
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25 | (8) |
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Hemoglobin E-β-Thalassemia: Progress Report from the International Study Group |
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33 | (7) |
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Survival and Complications in Thalassemia |
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40 | (8) |
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Part II. Gene Regulation and Therapy |
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Role of Intergenic Human γ-δ-Globin Sequences in Human Hemoglobin Switching and Reactivation of Fetal Hemoglobin in Adult Erythroid Cells |
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48 | (7) |
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Isolation and Characterization of Hematopoietic Transcription Factor Complexes by in Vivo Biotinylation Tagging and Mass Spectrometry |
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55 | (13) |
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Germline Epimutation: A Basis for Epigenetic Disease in Humans |
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68 | (10) |
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Progress Toward the Genetic Treatment of the β-Thalassemias |
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78 | (14) |
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Understanding α-Globin Gene Regulation: Aiming to Improve the Management of Thalassemia |
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92 | (11) |
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Role of Alpha Hemoglobin-Stabilizing Protein in Normal Erythropoiesis and β-Thalassemia |
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103 | (15) |
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Part III. Mechanisms of Cell Injury from Iron Overload in Thalassemia |
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Role of Iron in Inducing Oxidative Stress in Thalassemia: Can It Be Prevented by Inhibition of Absorption and by Antioxidants? |
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118 | (6) |
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Objectives and Mechanism of Iron Chelation Therapy |
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124 | (12) |
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Ferritin as an Iron Concentrator and Chelator Target |
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136 | (5) |
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Part IV. Iron Chelator: Development and Therapy |
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The Design of Orally Active Iron Chelators |
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141 | (14) |
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Recent Insights into Interactions of Deferoxamine with Cellular and Plasma Iron Pools: Implications for Clinical Use |
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155 | (14) |
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169 | (6) |
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Combined Therapy with Deferroxamine and Deferiprone |
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175 | (8) |
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Evaluation of ICL670, a Once-Daily Oral Iron Chelator in a Phase III Clinical Trial of β-Thalassemia Patients with Transfusional Iron Overload |
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183 | (3) |
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Part V. New Advances in Stem Cell Transplantation |
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Unrelated Bone Marrow Transplantation for β-Thalassemia Patients: The Experience of the Italian Bone Marrow Transplant Group |
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186 | (10) |
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Bone Marrow Transplantation in Adults with Thalassemia: Treatment and Long-Term Follow-Up |
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196 | (10) |
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Sibling Donor Cord Blood Transplantation for Thalassemia Major: Experience of the Sibling Donor Cord Blood Program |
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206 | (8) |
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Elizabeth T. Trachtenberg |
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Stem Cell Transplantation with S-59 Photochemically Treated T-Cell Add-Backs to Establish Allochimerism in Murine Thalassemia |
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214 | (9) |
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Preimplantation Genetics: Improving Access to Stem Cell Therapy |
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223 | (5) |
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Part VI. New Therapy for Thalassemia |
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Pharmacological Induction of Fetal Hemoglobin: Why Haven't We Been More Successful in Thalassemia? |
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228 | (10) |
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Successful Correction of the Human Cooley's Anemia β-Thalassemia Major Phenotype Using a Lentiviral Vector Flanked by the Chicken Hypersensitive Site 4 Chromatin Insulator |
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238 | (12) |
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Single and Combination Drug Therapy for Fetal Hemoglobin Augmentation in Hemoglobin E-β0-Thalassemia: Considerations for Treatment |
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250 | (7) |
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Induction of Fetal Globin in β-Thalassemia: Cellular Obstacles and Molecular Progress |
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257 | (9) |
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Part VII. Barriers to Optimal Survival in Thalassemia |
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Caring for Adults with Thalassemia in a Pediatric World |
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266 | (7) |
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Quality of Life in Thalassemia |
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273 | (10) |
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Thalassemia and Learning: Neurocognitive Function in Children |
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283 | (7) |
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Treatment of Hepatitis C Virus Infection in Thalassemia |
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290 | (10) |
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Fertility and Pregnancy in Thalassemia Major |
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300 | (8) |
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Part VIII. Emerging Issues in Thalassemia |
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A Phase I/II Clinical Trial of β-Globin Gene Therapy for β-Thalassemia |
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308 | (9) |
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Coagulation and Splenectomy: An Overview |
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317 | (8) |
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Transfusion Safety: Where Are We Today? |
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325 | (17) |
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Pulmonary Hypertension in β-Thalassemia |
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342 | (8) |
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Part IX. Noninvasive Iron Measurements |
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Monitoring Long-Term Efficacy of Iron Chelation Treatment with Biomagnetic Liver Susceptometry |
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350 | (8) |
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Methods for Noninvasive Measurement of Tissue Iron in Cooley's Anemia |
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358 | (15) |
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T2* Magnetic Resonance and Myocardial Iron in Thalassemia |
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373 | (6) |
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Measurement and Mapping of Liver Iron Concentrations Using Magnetic Resonance Imaging |
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379 | (7) |
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Physiology and Pathophysiology of Iron Cardiomyopathy in Thalassemia |
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386 | (10) |
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Eighth Cooley's Anemia Symposium: Summation and Perspective |
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396 | (11) |
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Rescued Mice with Hb E Transgene--Developed Red Cell Changes Similar to Human β-Thalassemia/HbE Disease |
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407 | (10) |
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Exploring the Role of Hepcidin, an Antimicrobial and Iron Regulator Peptide, in Increased Iron Absorption in β-Thalassemia |
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417 | (6) |
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Murine and Math Models for the Level of Stable Mixed Chimerism to Cure β-Thalassemia by Nonmyeloablative Bone Marrow Transplantation |
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423 | (6) |
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Non-Transferrin-Bound Iron During Blood Transfusion Cycles in β-Thalassemia Major |
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429 | (4) |
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Genetic Analysis of Candidate Modifier Polymorphisms in HbE-β0-Thalassemia Patients |
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433 | (6) |
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Measuring Chromosome Breaks in Patients with Thalassemia |
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439 | (6) |
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Longitudinal Study of Survival and Causes of Death in Patients with Thalassemia Major in Greece |
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445 | (6) |
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Osteoporosis in β-Thalassemia: Clinical and Genetic Aspects |
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451 | (6) |
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Quality of Life in Patients with Thalassemia Intermedia Compared to Thalassemia Major |
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457 | (5) |
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Low Bone Mineral Density in Adolescents with β-Thalassemia |
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462 | (5) |
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Pulmonary Hypertension and Cardiopulmonary Disease |
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Does Heterozygous β-Thalassemia Confer a Protection against Coronary Artery Disease? |
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467 | (4) |
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Therapeutic Approaches to Pulmonary Hypertension in Hemoglobinopathies: Efficacy and Safety of Sildenafil in the Treatment of Severe Pulmonary Hypertension in Patients with Hemoglobinopathy |
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471 | (5) |
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Utility of Holter Electrocardiogram in Iron-Overloaded Hemoglobinopathies |
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476 | (5) |
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Hemolysis-Associated Pulmonary Hypertension in Thalassemia |
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481 | (5) |
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Transfusion, Iron Overload, and Therapy |
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A Simple Model to Assess and Improve Adherence to Iron Chelation Therapy with Deferoxamine in Patients with Thalassemia |
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486 | (6) |
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Preclinical and Clinical Development of Deferitrin, a Novel, Orally Available Iron Chelator |
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492 | (3) |
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Allosensitization in Patients Receiving Multiple Blood Transfusions |
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495 | (5) |
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Prenatal Diagnosis and Treatment |
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Outcomes of Preimplantation Genetic Diagnosis Therapy in Treatment of β-Thalassemia: A Retrospective Analysis |
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500 | (4) |
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Separation of Normoblasts from Whole Blood by Ultracentrifugation on Arabino---Galactane Discontinuous Gradients: A Tool for Prenatal Diagnosis |
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504 | (3) |
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Prenatal Diagnosis of Hemoglobinopathies in Ontario, Canada |
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507 | (4) |
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The Splenectomy Controversy |
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511 | (3) |
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Diagnosis and Management of Alpha Thalassemia Disorders |
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514 | (2) |
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Management of the ``Difficult'' Iron-Overloaded Patient |
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516 | (2) |
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Genotypes and Phenotypes of Thalassemia: A Discussion |
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518 | (4) |
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Nutrition: Role in Health and Therapy |
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522 | (1) |
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| Index of Contributors |
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