Gene and Cell Therapies for Beta-Globinopathies

  • Punam Malik
  • John Tisdale

Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 1013)

Also part of the American Society of Gene & Cell Therapy book sub series (ASGCT, volume 1013)

Table of contents

  1. Front Matter
    Pages i-xi
  2. Patrick T. McGann, Alecia C. Nero, Russell E. Ware
    Pages 1-26
  3. Françoise Bernaudin, Corinne Pondarré, Claire Galambrun, Isabelle Thuret
    Pages 89-122
  4. Courtney D. Fitzhugh, Allistair Abraham, Matthew M. Hsieh
    Pages 123-153
  5. Alisa C. Dong, Stefano Rivella
    Pages 155-176
  6. Matthew H. Porteus
    Pages 203-217
  7. Back Matter
    Pages 241-248

About this book


This book provides a comprehensive review of gene and cell therapy approaches for hemoglobinopathies. It covers the progress and current state of the field in hematopoietic stem cell transplantation for hemoglobinopathies and genetic correction of autologous hematopoietic stem cells. Chapters focus on such topics as the current standards of care and long term outcomes of b-thalassemias and sickle cell disease, the ability to generate autologous embryonic stem cell-like cells from primary somatic cells, and genetic strategies to reactivate fetal hemoglobin production. In addition to offering a thorough review of the field in its present state, this book also sheds light on the major changes expected in coming years.


Gene and Cell Therapies for Beta-Globinopathies is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy.


Beta-Globinopathies Hemoglobinopathies cell therapy gene therapy thalassemias

Editors and affiliations

  • Punam Malik
    • 1
  • John Tisdale
    • 2
  1. 1.Cincinnati Children’s HospitalComprehensive Sickle Cell ProgramCincinnatiUSA
  2. 2.National Institutes of Health and Human ServicesBethesdaUSA

Bibliographic information

  • DOI
  • Copyright Information Springer Science+Business Media LLC 2017
  • Publisher Name Springer, New York, NY
  • eBook Packages Biomedical and Life Sciences
  • Print ISBN 978-1-4939-7297-5
  • Online ISBN 978-1-4939-7299-9
  • Series Print ISSN 0065-2598
  • Series Online ISSN 2214-8019
  • Buy this book on publisher's site