List of contributors ix Foreword xiii Preface xv 1 Beginnings: the molecular pathology of hemoglobin 1 Douglas Higgs and Mohsin Badat 2 Stem cells 17 David T. Scadden 3 The genetics of acute myeloid leukemia 33 Amye M. Harrigan and Amy M. Trottier 4 Molecular diagnostics and risk assessment in myeloid malignancies 51 Christian Scharenberg and Torsten Haferlach 5 Molecular basis of acute lymphoblastic leukemia 67 Shunsuke Kimura and Charles G. Mullighan 6 Chronic myelogenous leukemia 83 Hagop Kantarjian, Elias Jabbour and Susan O’Brien 7 Myeloproliferative neoplasms 99 Jessica Spiers, Dina Mahdi, Christopher Saunders and Donal P. McLornan 8 Lymphoma genetics 113 Giovanni Insuasti- Beltran, Chad M. McCall and Eric D. Hsi 9 Molecular basis of chronic lymphocytic leukemia 127 Bita Fakhri and Alexey Danilov 10 The molecular biology of multiple myeloma 137 Udit Yadav and Wilson I. Gonsalves 11 The molecular basis of bone marrow failure syndromes and red cell enzymopathies 145 Deena Iskander, Lucio Luzzatto and Anastasios Karadimitris 12 Anemia of chronic disease 169 Tomas Ganz and Elizabeta Nemeth 13 Molecular basis of iron metabolism 175 Jodie L. Babitt and Tomas Ganz 14 Perspectives in genomics and sickle cell disease therapeutics 187 Ambroise Wonkam 15 Molecular coagulation and thrombophilia 201 Björn Dahlbäck and Andreas Hillarp 16 The molecular basis of hemophilia 217 Keith Gomez 17 The molecular basis of von Willebrand disease 231 Omid Seidizadeh and Luciano Baronciani 18 Platelet disorders 251 Kenneth J. Clemetson 19 The molecular basis of blood cell alloantigens 267 Winnie Chong, Louise Tilley and Colin Brown 20 Functions of blood group antigens 285 Jonathan S. Stamler, Marilyn J. Telen and Carla Dinardo 21 Autoimmune hematological disorders 299 Drew Provan and John W. Semple 22 Molecular therapeutics in hematology: gene therapy 321 William M. McKillop and Jeffrey A. Medin 23 Pharmacogenomics 343 Leo Kager and William E. Evans 24 Cancer stem cells 359 Sara Ali and Dominique Bonnet 25 CAR- T cell therapy 369 Jude Franklin, Prateek Pophali, Zachary Jackson, David Wald and David Avigan 26 Molecular basis of transplantation 397 Pramila Krishnamurthy, Victoria Potter and Francesco Dazzi Index 413

Provides up-to-date and reliable information on the molecular basis of major hematologic diseases, authored and edited by leading experts in the field Molecular techniques play a central role in the diagnosis and treatment of blood diseases. Molecular characterization of genes and proteins has improved our understanding of the pathogeneses of hematologic disorders and led to the development of exciting new drug therapies and recombinant proteins. Molecular Hematology provides a core knowledgebase on the molecular basis of major hematologic diseases. Perfect for those with limited exposure to hematologic molecular biology, this accessible volume demonstrates the clinical relevance of molecular biology in the diagnosis and treatment of blood disorders such as hemophilia, anemia, multiple myeloma, thrombophilia, platelet disorders, leukemia, and lymphoma. Now in its fifth edition, Molecular Hematology is fully revised to incorporate current hematology research and practice. Entirely new chapters cover topics such as CAR-T cell technology, cancer cell biology, gene therapy and gene editing in clinical practice, and the impact of molecular research on the diagnosis and treatment of various malignant hematologic diseases. This edition includes updated suggested readings and a wealth of new full-color charts, graphs, and illustrations throughout. Including access to a companion Digital Edition providing interactive search, downloadable illustrations, and notation tools, Molecular Hematology, Fifth Edition, is an invaluable resource for established and trainee clinicians, clinical scientists and researchers, medical students, and upper-level undergraduates in the fields of hematology, oncology, and molecular biology.