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Viral Vectors for Gene Therapy

Methods and Protocols

  • Book
  • © 2019

Overview

Editors:
  1. Fredric P. Manfredsson

    1. Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, USA

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  2. Matthew J. Benskey

    1. Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, USA

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  • Includes cutting-edge methods and protocols
  • Provides step-by-step detail essential for reproducible results
  • Contains key notes and implementation advice from the experts

Part of the book series: Methods in Molecular Biology (MIMB, volume 1937)

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Table of contents (21 protocols)

  1. Front Matter

    Pages i-xiv
    Download chapter PDF

  2. Introduction

    1. Front Matter

      Pages 1-1
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    2. Basic Concepts in Viral Vector-Mediated Gene Therapy

      • Matthew J. Benskey, Ivette M. Sandoval, Kathryn Miller, Rhyomi L. Sellnow, Aysegul Gezer, Nathan C. Kuhn et al.
      Pages 3-26

  3. Novel Modes of Gene Therapy (Going Beyond Overexpression and Knockdown)

    1. Front Matter

      Pages 27-27
      Download chapter PDF

    2. Design and Assembly of CRISPR/Cas9 Lentiviral and rAAV Vectors for Targeted Genome Editing

      • Ivette M. Sandoval, Timothy J. Collier, Fredric P. Manfredsson
      Pages 29-45

    3. Design, Construction, and Application of Transcription Activation-Like Effectors

      • Peter Deng, Sakereh Carter, Kyle Fink
      Pages 47-58

    4. Practical Considerations for the Use of DREADD and Other Chemogenetic Receptors to Regulate Neuronal Activity in the Mammalian Brain

      • Patrick Aldrin-Kirk, Tomas Björklund
      Pages 59-87

  4. Viral Vectors

    1. Front Matter

      Pages 89-89
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    2. AAV Production Using Baculovirus Expression Vector System

      • Quentin Sandro, Karima Relizani, Rachid Benchaouir
      Pages 91-99

    3. Multimodal Production of Adeno-Associated Virus

      • Ivette M. Sandoval, Nathan M. Kuhn, Fredric P. Manfredsson
      Pages 101-124

    4. Generation of High-Titer Pseudotyped Lentiviral Vectors

      • Shuang Hu, Mingjie Li, Ramesh Akkina
      Pages 125-134

    5. A Scalable Lentiviral Vector Production and Purification Method Using Mustang Q Chromatography and Tangential Flow Filtration

      • Stuart Tinch, Kathy Szczur, William Swaney, Lilith Reeves, Scott R. Witting
      Pages 135-153

    6. Current Use of Adenovirus Vectors and Their Production Methods

      • Ekramy E. Sayedahmed, Rashmi Kumari, Suresh K. Mittal
      Pages 155-175

    7. Construction of Oncolytic Herpes Simplex Virus with Therapeutic Genes of Interest

      • Andranik Kahramanian, Toshihiko Kuroda, Hiroaki Wakimoto
      Pages 177-188

    8. Poxviruses as Gene Therapy Vectors: Generating Poxviral Vectors Expressing Therapeutic Transgenes

      • Steven J. Conrad, Jia Liu
      Pages 189-209

  5. Viral Vector Delivery

    1. Front Matter

      Pages 211-211
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    2. AAV-Mediated Gene Delivery to the Mouse Liver

      • Sharon C. Cunningham, Ian E. Alexander
      Pages 213-219

    3. Surgical Methods for Inner Ear Gene Delivery in Neonatal Mouse

      • Kevin Isgrig, Wade W. Chien
      Pages 221-226

    4. Gene Transfer to Mouse Kidney In Vivo

      • C. J. Rocca, S. Cherqui
      Pages 227-234

    5. Co-Delivery of a Short-Hairpin RNA and a shRNA-Resistant Replacement Gene with Adeno-Associated Virus: An Allele-Independent Strategy for Autosomal-Dominant Retinal Disorders

      • Michael T. Massengill, Brianna M. Young, Alfred S. Lewin, Cristhian J. Ildefonso
      Pages 235-258
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Keywords

About this book

This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. 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 tips on troubleshooting and avoiding known pitfalls.

Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.


Editors and Affiliations

  • Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, USA

    Fredric P. Manfredsson, Matthew J. Benskey

Bibliographic Information

  • Book Title: Viral Vectors for Gene Therapy

  • Book Subtitle: Methods and Protocols

  • Editors: Fredric P. Manfredsson, Matthew J. Benskey

  • Series Title: Methods in Molecular Biology

  • DOI: https://doi.org/10.1007/978-1-4939-9065-8

  • Publisher: Humana New York, NY

  • eBook Packages: Springer Protocols

  • Copyright Information: Springer Science+Business Media, LLC, part of Springer Nature 2019

  • Hardcover ISBN: 978-1-4939-9064-1Published: 01 February 2019

  • eBook ISBN: 978-1-4939-9065-8Published: 31 January 2019

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XIV, 328

  • Number of Illustrations: 13 b/w illustrations, 33 illustrations in colour

  • Topics: Gene Therapy, Virology

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