Genetic Modification of Hematopoietic Stem Cells Methods and Protocols 1st Edition by Guillermo Guenechea, Christopher Baum, Jose Segovia ISBN 9781588299802, 1588299805

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ISBN 10:  1588299805 

ISBN 13: 9781588299802

Author: Guillermo Guenechea,  Christopher Baum, Jose C. Segovia
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).

Table of contents: 

1. Immunomagnetic Enrichment of Human and Mouse Hematopoietic Stem Cells for Gene Therapy Applications Guillermo Guenechea, Jose C. Segovia, and Juan A. Bueren

2. Isolation of Human and Mouse Hematopoietic Stem Cells Yuk Yin Ng, Miranda R.M. Baert, Edwin F.E. de Haas, Karin Pike-Overzet, and Frank J.T. Staal

3. Murine Hematopoietic Stem Cell Transduction Using Retroviral Vectors Ute Modlich, Axel Schambach, Zhixiong Li, and Bernhard Schiedlmeier

4. Genetic Modification of Human Hematopoietic Cells:

Preclinical Optimization of Oncoretroviral-mediated Gene Transfer for Clinical Trials Tulin Budak-Alpdogan and Isabelle Rivière

5. Short-Term Culture of Human CD34+ Cells for Lentiviral Gene Transfer. Francesca Santoni de Sio and Luigi Naldini

6. T Cell Culture for Gammaretroviral Transfer Sebastian Newrzela, Gunda Brandenburg, and Dorothee von Laer

7. Retroviral Transduction of Murine Primary T Lymphocytes. James Lee, Michel Sadelain, and Renier Brentjens

8. Lentiviral Vector Gene Transfer into Human T Cells.. Els Verhoeyen, Caroline Costa, and Francois-Loic Cosset

9. DNA Transposons for Modification of Human Primary T Lymphocytes.

Xin Huang, Andrew Wilber, R. Scott McIvor, and Xianzheng Zhou

10. Retroviral Gene Transfer into Primary Human Natural Killer Cells Evren Alici, Tolga Sutlu, and M. Sirac Dilber

11. Lentiviral Vector-Mediated Genetic Programming of Mouse and Human Dendritic Cells. Renata Stripecke

12. In Situ (In Vivo) Gene Transfer into Murine Bone Marrow Stem Cells Dao Pan

13. In Vivo and Ex Vivo Gene Transfer in Thymocytes and Thymocyte Precursors.

Oumeya Adjali, Amélie Montel-Hagen, Louise Swainson, Sophie Marty, Rita Vicente, Cedric Mongellaz, Chantal Jacquet, Valérie Zimmermann, and Naomi Taylor

14. Design and Production of Retro- and Lentiviral Vectors for Gene Expression in Hematopoietic Cells Axel Schambach, William P. Swaney, and Johannes C.M. van der Loo

15. Knock-Down of Gene Expression in Hematopoietic Cells Michaela Scherr, Letizia Venturini, and Matthias Eder

16. The Use of Retroviral Vectors for tet-Regulated Gene Expression in Cell Populations. Rainer Löw

17. Detection of Replication Competent Retrovirus and Lentivirus. Lakshmi Sastry and Kenneth Cornetta

18. Release Testing of Retroviral Vectors and Gene-Modified Cells Diana Nordling, Anne Kaiser, and Lilith Reeves

19. Copy Number Determination of Genetically-Modified Hematopoietic Stem Cells. Todd Schuesler, Lilith Reeves, Christof von Kalle, and Elke Grassman

20. Tissue Procurement for Molecular Studies Using Laser-Assisted Microdissection. Ulrich Lehmann and Hans Kreipe

21. Leukemia Diagnosis in Murine Bone Marrow Transplantation Models.. Zhixiong Li, Ute Modlich, and Anjali Mishra

22. Humanized Mouse Models to Study the Human Haematopoietic Stem Cell Compartment Dominique Bonnet

23. Canine Models of Gene-Modified Hematopoiesis Brian C. Beard and Hans-Peter Kiem

24. Detection of Retroviral Integration Sites by Linear Amplification-Mediated PCR and Tracking of Individual Integration Clones in Different Samples Manfred Schmidt, Kerstin Schwarzwaelder,

Cynthia C. Bartholomae, Hanno Glimm, and Christof von Kalle

25. Retroviral Insertion Site Analysis in Dominant Haematopoietic Clones

Olga S. Kustikova, Ute Modlich, and Boris Felse

26. Tracking Gene-Modified T Cells In Vivo. Alessandra Recchia and Fulvio Mavilio

27. DNA Microarray Studies of Hematopoietic Subpopulations. Karin Pike-Overzet, Dick de Ridder, Tom Schonewille, and Frank J.T. Staal

28. Quantification of Genomic Mutations in Murine Hematopoietic Cells Hartmut Geiger, David Schleimer, Kalpana J. Nattamai, and Jan Vijg

29. Proteomics Studies After Hematopoietic Stem Cell Transplantation……. Eva M. Weissinger, Petra Zürbig, and Arnold Ganser

30. Spectral Karyotyping and Fluorescence In Situ Hybridization of Murine Cells

Cornelia Rudolph and Brigitte Schlegelberger

31. Database Setup for Preclinical Studies of Gene-Modified Hematopoiesis

Brenden Balcik, Elke Grassman, and Lilith Reeves

32. The US and EU Regulatory Perspectives on the Clinical

Use of Hematopoietic Stem/Progenitor Cells Genetically

Modified Ex Vivo by Retroviral Vectors..

Carolyn A. Wilson and Klaus Cichutek

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Tags: Guillermo Guenechea, Christopher Baum, Jose Segovia, Genetic Modification, Hematopoietic Stem