Gene Therapy for Neurological Disorders

Methods and Protocols

  • Fredric P. Manfredsson

Part of the Methods in Molecular Biology book series (MIMB, volume 1382)

Table of contents

  1. Front Matter
    Pages i-xiv
  2. Introduction

  3. Expression Cassettes

    1. Front Matter
      Pages 19-19
    2. Matthew L. Hirsch, Sonya J. Wolf, R. J. Samulski
      Pages 21-39
    3. Ludivine Breger, Erika Elgstrand Wettergren, Luis Quintino, Cecilia Lundberg
      Pages 57-66
    4. Gabriela Toro Cabrera, Christian Mueller
      Pages 67-80
    5. Sebastian Kügler
      Pages 81-91
  4. Viral Vector Production

    1. Front Matter
      Pages 93-93
    2. Corinna Burger, Kevin R. Nash
      Pages 95-106
    3. Matthew J. Benskey, Fredric P. Manfredsson
      Pages 107-114
    4. 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. Michael J. Castle, Heikki T. Turunen, Luk H. Vandenberghe, John H. Wolfe
      Pages 133-149
    3. Damien Marsic, Sergei Zolotukhin
      Pages 151-173
    4. Kenta Kobayashi, Shigeki Kato, Ken-ichi Inoue, Masahiko Takada, Kazuto Kobayashi
      Pages 175-186
    5. Jason G. Smith
      Pages 187-196
  6. Delivery Methods

    1. Front Matter
      Pages 197-197
    2. Ernesto A. Salegio, John Bringas, Krystof S. Bankiewicz
      Pages 217-230
    3. Sara E. Gombash, Kevin D. Foust
      Pages 231-237
    4. Ji-Yoen Kim, Stacy D. Grunke, Joanna L. Jankowsky
      Pages 239-250
    5. Hongwei Yu, Gregory Fischer, Quinn H. Hogan
      Pages 251-261
    6. Matthew J. Benskey, Fredric P. Manfredsson
      Pages 263-274
    7. Bradley Hammond, David L. Kreulen
      Pages 275-283
    8. Xue-Feng Ding, Ming Fan
      Pages 297-305
  7. Gene Therapy Based Modeling of Neurodegenerative Disorders

    1. Front Matter
      Pages 337-337
    2. Benjamin Combs, Andrew Kneynsberg, Nicholas M. Kanaan
      Pages 339-366
    3. D. Luke Fischer, Sara E. Gombash, Christopher J. Kemp, Fredric P. Manfredsson, Nicole K. Polinski, Megan F. Duffy et al.
      Pages 367-382
  8. Gene Therapy for the Treatment of Neurological Disorders

    1. Front Matter
      Pages 397-397
    2. Zachary T. McEachin, Anthony Donsante, Nicholas Boulis
      Pages 399-408
    3. Neha Kamran, Marianela Candolfi, Gregory J. Baker, Mariela Moreno Ayala, Marta Dzaman, Pedro R. Lowenstein et al.
      Pages 467-482
  9. Clinical Trials

    1. Front Matter
      Pages 483-483
  10. Back Matter
    Pages 491-493

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.



Gene Therapy Germline Manipulations Neurodegenerative Disorders Neurological Disorders Preclinical Transgene Cassettes

Editors and affiliations

  • Fredric P. Manfredsson
    • 1
  1. 1.Translational Science and Molecular MedicineMichigan State University, College of Human ScienceGrand RapidsUSA

Bibliographic information

  • DOI
  • Copyright Information Springer Science+Business Media New York 2016
  • Publisher Name Humana Press, New York, NY
  • eBook Packages Springer Protocols
  • Print ISBN 978-1-4939-3270-2
  • Online ISBN 978-1-4939-3271-9
  • Series Print ISSN 1064-3745
  • Series Online ISSN 1940-6029
  • Buy this book on publisher's site