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Gene and Cell Therapies for Beta-Globinopathies

  • Book
  • © 2017

Overview

Editors:
  1. Punam Malik

    1. Cincinnati Children’s Hospital, Comprehensive Sickle Cell Program, Cincinnati, USA

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    You can also search for this editor in PubMed   Google Scholar

  2. John Tisdale

    1. National Institutes of Health and Human Services, Bethesda, USA

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    You can also search for this editor in PubMed   Google Scholar

  • This book offers a combined review of state of the art in gene and cell therapies for hemoglobinopathies
  • Short, succinct and comprehensive review of the current state of the field
  • Prospects of gene and cell therapies in the next decade
  • Includes supplementary material: sn.pub/extras

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

Part of the book sub series: American Society of Gene & Cell Therapy (ASGCT)

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Table of contents (9 chapters)

  1. Front Matter

    Pages i-xi
    Download chapter PDF

  2. Clinical Features of β-Thalassemia and Sickle Cell Disease

    • Patrick T. McGann, Alecia C. Nero, Russell E. Ware
    Pages 1-26

  3. Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease

    • Swee Lay Thein
    Pages 27-57

  4. Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease

    • Satheesh Chonat, Charles T. Quinn
    Pages 59-87

  5. Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia

    • Françoise Bernaudin, Corinne PondarrĂ©, Claire Galambrun, Isabelle Thuret
    Pages 89-122

  6. Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease

    • Courtney D. Fitzhugh, Allistair Abraham, Matthew M. Hsieh
    Pages 123-153

  7. Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia

    • Alisa C. Dong, Stefano Rivella
    Pages 155-176

  8. Reactivation of Fetal Hemoglobin for Treating β-Thalassemia and Sickle Cell Disease

    • Shuaiying Cui, James Douglas Engel
    Pages 177-202

  9. Genome Editing for the β-Hemoglobinopathies

    • Matthew H. Porteus
    Pages 203-217

  10. Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cells (iPSCs): The Next Frontier

    • Eirini P. Papapetrou
    Pages 219-240

  11. Back Matter

    Pages 241-248
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Keywords

About this book

Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, which are often available only to 15-20% of patients. An alternative to allogeneic HS

CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances.

Editors and Affiliations

  • Cincinnati Children’s Hospital, Comprehensive Sickle Cell Program, Cincinnati, USA

    Punam Malik

  • National Institutes of Health and Human Services, Bethesda, USA

    John Tisdale

About the editors

Punam Malik received her M.D. degree from Lady Hardinge Medical College, University of Delhi, India.  She completed a pediatric residency at Kalawati Saran Children;s Hospital, Delhi and at Children’s Hospital Los Angeles, Los Angeles, California, and then then trained in hematology-oncology at Children’s Hospital Los Angeles, where she served as fellow, Assistant Professor from 1999-2005 and a tenured Associate Professor in 2006 at CHLA, University of Southern California.  She joined Cincinnati Children’s Hospital Medical Center and the University of Cincinnati in 2007 as the Program Director of the Gene and Cell Therapy Program and in 2008, she became the director of the Translational Core Laboratories at CCHMC. In 2010, she became a Professor of Pediatrics.  In 2012, she became the director of the Cincinnati Comprehensive Sickle Cell Center, and in 2014 she became the Marjorie Johnson Endowed Professor of Gene and Cell Therapy.  Her group focuses on the pathobiology of sickle cell disease and thalassemia, identifying mechanisms of organ pathologies in sickle cell disease, and developing curative strategies for hemoglobinopathies and other hematopoietic stem cell disorders through transplantation of genetically modified autologous bone marrow stem cells.  

John Tisdale received his M.D. degree from the Medical University of South Carolina in Charleston.  He completed an internal medicine and chief residency at Vanderbilt University Medical Center in Nashville and then trained in hematology in the Hematology Branch, National Heart, Lung and Blood Institute (NHLBI), where he served as a postdoctoral fellow.  He joined the Molecular and Clinical Hematology Branch of NHLBI in 1998 and is now the Chief of the Cellular and Molecular Therapeutics Section.  His group focuses on developing curative strategies for sickle cell disease through transplantation of allogeneic or genetically modified autologous bone marro


w stem cells.  

Bibliographic Information

  • Book Title: Gene and Cell Therapies for Beta-Globinopathies

  • Editors: Punam Malik, John Tisdale

  • Series Title: Advances in Experimental Medicine and Biology

  • DOI: https://doi.org/10.1007/978-1-4939-7299-9

  • Publisher: Springer New York, NY

  • eBook Packages: Biomedical and Life Sciences, Biomedical and Life Sciences (R0)

  • Copyright Information: Springer Science+Business Media LLC 2017

  • Hardcover ISBN: 978-1-4939-7297-5Published: 20 November 2017

  • Softcover ISBN: 978-1-4939-8446-6Published: 28 August 2018

  • eBook ISBN: 978-1-4939-7299-9Published: 09 November 2017

  • Series ISSN: 0065-2598

  • Series E-ISSN: 2214-8019

  • Edition Number: 1

  • Number of Pages: XII, 248

  • Number of Illustrations: 4 b/w illustrations, 12 illustrations in colour

  • Topics: Gene Therapy, Cell Biology

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