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Adeno-Associated Virus Vectors

Design and Delivery

  • Book
  • © 2019

Overview

Editors:
  1. Michael J. Castle

    1. Department of Neurosciences, University of California, San Diego, La Jolla, USA

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

Part of the book series: Methods in Molecular Biology (MIMB, volume 1950)

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Table of contents (24 protocols)

  1. Front Matter

    Pages i-xii
    Download chapter PDF

  2. AAV Vector Design

    1. Front Matter

      Pages 1-1
      Download chapter PDF

    2. Design of AAV Vectors for Delivery of RNAi

      • Florie Borel, Christian Mueller
      Pages 3-18

    3. Design of AAV Vectors for Delivery of Large or Multiple Transgenes

      • Aman Patel, Junling Zhao, Dongsheng Duan, Yi Lai
      Pages 19-33

    4. Ligand Coupling to the AAV Capsid for Cell-Specific Gene Transfer

      • Johanna Reul, Alexander Muik, Christian J. Buchholz
      Pages 35-50

    5. Quantitative and Digital Droplet-Based AAV Genome Titration

      • Julio Sanmiguel, Guangping Gao, Luk H. Vandenberghe
      Pages 51-83

    6. Single-Stranded DNA Virus Sequencing (SSV-Seq) for Characterization of Residual DNA and AAV Vector Genomes

      • Emilie Lecomte, Adrien Leger, Magalie Penaud-Budloo, Eduard Ayuso
      Pages 85-106

    7. In Situ Hybridization for Detection of AAV-Mediated Gene Expression

      • Jacqueline E. Hunter, Brittney L. Gurda, Sea Young Yoon, Michael J. Castle, John H. Wolfe
      Pages 107-122

    8. Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina

      • Wenhan Yu, Zhijian Wu
      Pages 123-139

  3. AAV Delivery to the Central Nervous System

    1. Front Matter

      Pages 141-141
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    2. Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System

      • Dominic J. Gessler, Phillip W. L. Tai, Jia Li, Guangping Gao
      Pages 143-163

    3. Intraspinal and Intracortical Delivery of AAV Vectors for Intersectional Circuit Tracing in Non-transgenic Species

      • James M. Conner, Greg L. Bain, Jennifer N. Dulin
      Pages 165-176

    4. MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain

      • Zeinab Noroozian, Kristiana Xhima, Yuexi Huang, Brian K. Kaspar, Sebastian Kügler, Kullervo Hynynen et al.
      Pages 177-197

    5. AAV-Mediated Gene Delivery to the Spinal Cord by Intrathecal Injection

      • Cristina D. Peterson, Alexander G. J. Skorput, Kelley F. Kitto, George L. Wilcox, Lucy Vulchanova, Carolyn A. Fairbanks
      Pages 199-207

    6. Subpial AAV Delivery for Spinal Parenchymal Gene Regulation in Adult Mammals

      • Mariana Bravo-Hernández, Takahiro Tadokoro, Martin Marsala
      Pages 209-233

  4. AAV Delivery to Sensory Systems

    1. Front Matter

      Pages 235-235
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    2. Peripheral AAV Injection for Retrograde Transduction of Dorsal Root and Trigeminal Ganglia

      • David C. Bloom, Zachary L. Watson, Donna M. Neumann
      Pages 237-247

    3. SubILM Injection of AAV for Gene Delivery to the Retina

      • Paul D. Gamlin, John J. Alexander, Sanford L. Boye, C. Douglas Witherspoon, Shannon E. Boye
      Pages 249-262

    4. Intracameral Delivery of AAV to Corneal Endothelium for Expression of Secretory Proteins

      • Jeffrey O’Callaghan, Matthew Campbell, Peter Humphries
      Pages 263-270

    5. AAV-Mediated Gene Delivery to the Inner Ear

      • Omar Akil, Lawrence Lustig
      Pages 271-282
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Keywords

About this book

This volume provides a complete and timely guide to the use of adeno-associated virus (AAV)  vectors for genetic manipulation of mammalian tissues. Beginning with methods for the design and characterization of AAV vectors, the book continues with protocols for AAV delivery to various components of the central nervous system, to a number of sensory systems, and to a broad range of other tissues. Novel techniques such as ultrasound-targeted delivery to the brain, subpial delivery to the spinal cord, and subILM delivery to the retina are accompanied by chapters that provide an overview and comparison of current methods for AAV delivery to tissues such as brain, heart, liver, and lung. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, readily reproducible step-by-step laboratory protocols, and tips for troubleshooting and avoiding known pitfalls. 

Authoritative and comprehensive, Adeno-Associated Virus Vectors: Design and Delivery aims to enhance the utility of AAV vectors for targeted gene transfer to living animals and continue the ongoing development of novel AAV-based gene therapies for human disease.

Editors and Affiliations

  • Department of Neurosciences, University of California, San Diego, La Jolla, USA

    Michael J. Castle

Bibliographic Information

  • Book Title: Adeno-Associated Virus Vectors

  • Book Subtitle: Design and Delivery

  • Editors: Michael J. Castle

  • Series Title: Methods in Molecular Biology

  • DOI: https://doi.org/10.1007/978-1-4939-9139-6

  • Publisher: Humana New York, NY

  • eBook Packages: Springer Protocols

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

  • Hardcover ISBN: 978-1-4939-9138-9Published: 20 February 2019

  • eBook ISBN: 978-1-4939-9139-6Published: 19 February 2019

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XII, 426

  • Number of Illustrations: 12 b/w illustrations, 50 illustrations in colour

  • Topics: Human Genetics, Virology

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