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Genetic Modification of Hematopoietic Stem Cells

Methods and Protocols

  • Book
  • © 2009

Overview

  • Provides an easily accessible reference volume for genetic modification of hematopoietic cells using various state-of-the-art vector systems
  • Comprehensive guide on functional, phenotypic and genotypic screening of gene-modified cells written by leading researchers in the field
  • Contains helpful insights into preclinical data acquisition and documentation for gene therapy, including regulatory comments from the EU and US perspectives
  • Covers a wide range of cell-based and animal assay formats and systems

Part of the book series: Methods in Molecular Biology (MIMB, volume 506)

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Table of contents (32 protocols)

Keywords

About this book

Gene Transfer into Hematopoietic Cells: From Basic Science to Clinical Application Christopher Baum 1. The Potential of Gene Transfer into Hematopoietic Vectors with the potential for stable transgene integration are Cells widely used in basic hematology and clinical trials of gene me- cine. In basic research, both gain-of-function and loss-of-fu- tion situations of individual genes can be created by gene transfer, leading to a wide range of applications in developmental biology, stem cell biology, immunology, leukemia research, and human genetics. With the first evidence of successful modification of murine hematopoietic cells using retroviral gene vectors (1, 2), researchers have also explored the therapeutic potential of this approach. To date, the emerging discipline of gene therapy is a highly diversified field that offers entirely novel approaches to treat a great variety of human diseases (3). All hematopoietic cell types are of major interest in this context, since the modification of the hematopoietic stem cell population may potentially give rise to a completely transgenic hematopoiesis with the potential to cure genetic disorders or fight severe chronic infections, and the targeting of mature cells such as lymphocytes or antigen-p- senting dendritic cells offers all types of transient and semiper- nent modifications of the immune system. The unifying principle of gene medicine is the need to transfer complex nucleic acids cells that do not contribute to the germline (somatic cells).

Reviews

From the reviews:

"This book provides a unique and comprehensive resource of protocols for the genetic modification of various hematopoietic cell types and up-to-date procedures for molecular and systemic monitoring. … This book is a great help for the design of excellent research in basic hematology, oncology, genetics, and immunology, and also promote the implementation of investigator-driven clinical studies using gene-modified hematopoietic cells. The book represents an outstanding work, which should not be missed in all biomedical research laboratories dealing with gene therapy of hematopoietic cells." (C. Altaner, Neoplasma, June, 2009)

"This book provides a unique and comprehensive resource of protocols for the genetic modification of various hematopoietic cell types and up-to-date procedures for molecular and systemic monitoring. … This book is a great help for the design of excellent research in basic hematology, oncology, genetics, and immunology, and also promote the implementation of investigator-driven clinical studies using gene-modified hematopoietic cells. The book represents an outstanding work, which should not be missed in all biomedical research laboratories dealing with gene therapy of hematopoietic cells." (C. Altaner, Neoplasma, April, 2009)

“Haematopoietic stem cells are attractive targets for gene therapy. … Each chapter provides a brief review of the area, followed by a step-by-step guide to undertaking experiments and includes details of reagents required and a list of useful notes or comments of issues that may arise. Comprehensive reference citations provide sources of further information when required. The book should provide a road map for researchers, clinical investigators and regulators involved in modifying haematopoietic cells.”­­­ (Waseem Qasim, Human Genetics, Vol. 128, May, 2010)

Editors and Affiliations

  • Department of Experimental Hematology, Hannover Medical School, Hannover, Germany

    Christopher Baum

Bibliographic Information

  • Book Title: Genetic Modification of Hematopoietic Stem Cells

  • Book Subtitle: Methods and Protocols

  • Editors: Christopher Baum

  • Series Title: Methods in Molecular Biology

  • DOI: https://doi.org/10.1007/978-1-59745-409-4

  • Publisher: Humana Totowa, NJ

  • eBook Packages: Springer Protocols

  • Copyright Information: Humana Press 2009

  • Hardcover ISBN: 978-1-58829-980-2Published: 19 December 2008

  • Softcover ISBN: 978-1-61737-866-9Published: 20 January 2011

  • eBook ISBN: 978-1-59745-409-4Published: 26 December 2008

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XXII, 490

  • Number of Illustrations: 74 b/w illustrations, 10 illustrations in colour

  • Topics: Biochemistry, general, Cell Biology, Stem Cells, Gene Therapy

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