Genetic Modification of Hematopoietic Stem Cells
Methods and Protocols
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Beschreibung
With the incredible potential of gene transfer into hematopoietic stem cells, active research in this field has become critically important. In Genetic Modification of Hematopoietic Stem Cells: Methods and Protocols, leading scientists in the field provide a compendium of protocols which cover the subject comprehensively, from the purification and culture of various types of hematopoietic cells for subsequent genetic modification by vector development and technical issues of small and large scale vector production, to the complex issue of monitoring and biosafety studies related to gene-modified hematopoiesis. Written in the highly successful Methods in Molecular Biology™ series format, the chapters in this volume present brief introductions to the topic, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and Notes sections, which allow the experts to highlight tips on troubleshooting and avoiding known pitfalls.
Unique and cutting-edge, Genetic Modification of Hematopoietic Stem Cells: Methods and Protocols is an ideal, thorough resource to promote further research and the implementation of investigator-driven clinical studies using gene-modified hematopoietic cells.
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).
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
Autor*in
Christopher Baum
Themen in »Genetic Modification of Hematopoietic Stem Cells«
Complex nucleic acids DNA Gene medicine Gene transfer Hematology Hematopoiesis Microarray Mutagenesis PCR Proteomics Termination gene expression gene therapy genes hybridization
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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)
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