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Prenatal Gene Therapy

Concepts, Methods, and Protocols

  • Book
  • © 2012

Overview

Editors:
  1. Charles Coutelle

    1. National Heart & Lung Inst., Molecular & Cellular Medicine Sec., Imperial College London, London, United Kingdom

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    You can also search for this editor in PubMed   Google Scholar

  2. Simon N. Waddington

    1. Reader in Gene Transfer Technology, Gene Transfer Technology Group, University College London, London, United Kingdom

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  • Aims at applying gene therapy during pregnancy for the prevention of human disease
  • Provides step-by-step detail essential fro reproducible results
  • Contains key notes and implementation advice from the experts
  • Includes supplementary material: sn.pub/extras

Part of the book series: Methods in Molecular Biology (MIMB, volume 891)

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Table of contents (17 protocols)

  1. Front Matter

    Pages i-x
    Download chapter PDF

  2. The Concept of Prenatal Gene Therapy

    • Charles Coutelle, Simon N. Waddington
    Pages 1-7

  3. Candidate Diseases for Prenatal Gene Therapy

    • Anna L. David, Simon N. Waddington
    Pages 9-39

  4. Vector Systems for Prenatal Gene Therapy: Choosing Vectors for Different Applications

    • Charles Coutelle, Simon N. Waddington
    Pages 41-53

  5. Vector Systems for Prenatal Gene Therapy: Principles of Adenovirus Design and Production

    • Raul Alba, Andrew H. Baker, Stuart A. Nicklin
    Pages 55-84

  6. Vector Systems for Prenatal Gene Therapy: Principles of Retrovirus Vector Design and Production

    • Steven J. Howe, Anil Chandrashekran
    Pages 85-107

  7. Vector Systems for Prenatal Gene Therapy: Principles of Adeno-Associated Virus Vector Design and Production

    • Christopher J. Binny, Amit C. Nathwani
    Pages 109-131

  8. Vector Systems for Prenatal Gene Therapy: Principles of Non-viral Vector Design and Production

    • Suet Ping Wong, Orestis Argyros, Richard P. Harbottle
    Pages 133-167

  9. Use of Manipulated Stem Cells for Prenatal Therapy

    • Jessica L. Roybal, Pablo Laje, Jesse D. Vrecenak, Alan W. Flake
    Pages 169-181

  10. Animal Models for Prenatal Gene Therapy: Choosing the Right Model

    • Vedanta Mehta, Donald Peebles, Anna L. David
    Pages 183-200

  11. Animal Models for Prenatal Gene Therapy: Rodent Models for Prenatal Gene Therapy

    • Jessica L. Roybal, Masayuki Endo, Suzanne M. K. Buckley, Bronwen R. Herbert, Simon N. Waddington, Alan W. Flake
    Pages 201-218

  12. Animal Models for Prenatal Gene Therapy: The Sheep Model

    • Khalil N. Abi-Nader, Michael Boyd, Alan W. Flake, Vedanta Mehta, Donald Peebles, Anna L. David
    Pages 219-248

  13. Animal Models for Prenatal Gene Therapy: The Nonhuman Primate Model

    • Citra N. Mattar, Arijit Biswas, Mahesh Choolani, Jerry K. Y. Chan
    Pages 249-271

  14. Choice of Surrogate and Physiological Markers for Prenatal Gene Therapy

    • Juliette M. K. M. Delhove, Ahad A. Rahim, Tristan R. McKay, Simon N. Waddington, Suzanne M. K. Buckley
    Pages 273-290

  15. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Use of Large Animal Models with Relevance to Human Application

    • Vedanta Mehta, Khalil N. Abi-Nader, David Carr, Jacqueline Wallace, Charles Coutelle, Simon N. Waddington et al.
    Pages 291-328

  16. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Mouse Models for Developmental Aberrations and Inadvertent Germ Line Transmission

    • Charles Coutelle, Simon N. Waddington, Michael Themis
    Pages 329-340

  17. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Genotoxicity Analysis In Vitro and on Small Animal Models Ex Vivo and In Vivo

    • Michael Themis
    Pages 341-370

  18. Risks, Benefits and Ethical, Legal, and Societal Considerations for Translation of Prenatal Gene Therapy to Human Application

    • Charles Coutelle, Richard Ashcroft
    Pages 371-387

  19. Back Matter

    Pages 389-392
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Keywords

About this book

The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions.  In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for  the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly successful Methods in Molecular Biology™ series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls.

 

Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine.

Editors and Affiliations

  • National Heart & Lung Inst., Molecular & Cellular Medicine Sec., Imperial College London, London, United Kingdom

    Charles Coutelle

  • Reader in Gene Transfer Technology, Gene Transfer Technology Group, University College London, London, United Kingdom

    Simon N. Waddington

Bibliographic Information

  • Book Title: Prenatal Gene Therapy

  • Book Subtitle: Concepts, Methods, and Protocols

  • Editors: Charles Coutelle, Simon N. Waddington

  • Series Title: Methods in Molecular Biology

  • DOI: https://doi.org/10.1007/978-1-61779-873-3

  • Publisher: Humana Totowa, NJ

  • eBook Packages: Springer Protocols

  • Copyright Information: Springer Science+Business Media, LLC. 2012

  • Hardcover ISBN: 978-1-61779-872-6Published: 31 May 2012

  • Softcover ISBN: 978-1-4939-5911-2Published: 23 August 2016

  • eBook ISBN: 978-1-61779-873-3Published: 31 May 2012

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: X, 392

  • Number of Illustrations: 26 b/w illustrations, 7 illustrations in colour

  • Topics: Animal Genetics and Genomics, Human Genetics, Gene Therapy

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