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  • © 2016

Gene Therapy for Neurological Disorders

Methods and Protocols

  • 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 1382)

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Table of contents (32 protocols)

  1. Front Matter

    Pages i-xiv
  2. Introduction

    1. Front Matter

      Pages 1-1
  3. Expression Cassettes

    1. Front Matter

      Pages 19-19
    2. Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors

      • Matthew L. Hirsch, Sonya J. Wolf, R. J. Samulski
      Pages 21-39
    3. Regulated Gene Therapy

      • Ludivine Breger, Erika Elgstrand Wettergren, Luis Quintino, Cecilia Lundberg
      Pages 57-66
    4. Design of shRNA and miRNA for Delivery to the CNS

      • Gabriela Toro Cabrera, Christian Mueller
      Pages 67-80
    5. Tissue-Specific Promoters in the CNS

      • Sebastian Kügler
      Pages 81-91
  4. Viral Vector Production

    1. Front Matter

      Pages 93-93
    2. Small-Scale Recombinant Adeno-Associated Virus Purification

      • Corinna Burger, Kevin R. Nash
      Pages 95-106
    3. Lentivirus Production and Purification

      • Matthew J. Benskey, Fredric P. Manfredsson
      Pages 107-114
    4. Viral Vector Production: Adenovirus

      • Julius W. Kim, Ramin A. Morshed, J. Robert Kane, Brenda Auffinger, Jian Qiao, Maciej S. Lesniak
      Pages 115-130
  5. Viral Vector Tropism

    1. Front Matter

      Pages 131-131
    2. Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids

      • Michael J. Castle, Heikki T. Turunen, Luk H. Vandenberghe, John H. Wolfe
      Pages 133-149
    3. Altering Tropism of rAAV by Directed Evolution

      • Damien Marsic, Sergei Zolotukhin
      Pages 151-173
    4. Altering Entry Site Preference of Lentiviral Vectors into Neuronal Cells by Pseudotyping with Envelope Glycoproteins

      • Kenta Kobayashi, Shigeki Kato, Ken-ichi Inoue, Masahiko Takada, Kazuto Kobayashi
      Pages 175-186
    5. Directed Evolution of Adenoviruses

      • Jason G. Smith
      Pages 187-196
  6. Delivery Methods

    1. Front Matter

      Pages 197-197

About this book

This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate and what are the proven methods to treat these disorders in preclinical models? 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.

Authoritative and thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.

Editors and Affiliations

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

    Fredric P. Manfredsson

About the editor

Fredric P. Manfredsson, Ph.D. Assistant Professor Michigan State University Division of Translational Science & Molecular Medicine Van Andel Institute 333 Bostwick Ave NE Grand Rapids, MI 49503 Tel: 616-234-0968 Fax: 616-234-0990

Bibliographic Information

  • Book Title: Gene Therapy for Neurological Disorders

  • Book Subtitle: Methods and Protocols

  • Editors: Fredric P. Manfredsson

  • Series Title: Methods in Molecular Biology

  • DOI: https://doi.org/10.1007/978-1-4939-3271-9

  • Publisher: Humana New York, NY

  • eBook Packages: Springer Protocols

  • Copyright Information: Springer Science+Business Media New York 2016

  • Hardcover ISBN: 978-1-4939-3270-2Published: 26 November 2015

  • Softcover ISBN: 978-1-4939-4896-3Published: 23 August 2016

  • eBook ISBN: 978-1-4939-3271-9Published: 26 November 2015

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XIV, 493

  • Topics: Neurosciences, Human Genetics, Neurology

Buy it now

Buying options

eBook USD 89.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book USD 119.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book USD 169.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Other ways to access