Overview
- 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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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.Similar content being viewed by others
Keywords
Table of contents (9 chapters)
Editors and Affiliations
About the editors
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