Retinal Gene Therapy

Methods and Protocols

  • Camiel J.F. Boon
  • Jan Wijnholds

Part of the Methods in Molecular Biology book series (MIMB, volume 1715)

Table of contents

  1. Front Matter
    Pages i-xvi
  2. Retinal Gene Therapy Vector Production and Biological Assays In Vitro

  3. Assays for Gene Augmentation and Editing In Vivo on Rodent and Macaque Retina

    1. Front Matter
      Pages 99-99
    2. Jimmy de Melo, Seth Blackshaw
      Pages 101-111
    3. Sandy S. Hung, Fan Li, Jiang-Hui Wang, Anna E. King, Bang V. Bui, Guei-Sheung Liu et al.
      Pages 113-133
    4. C. Henrique Alves, Jan Wijnholds
      Pages 135-151
    5. Ivana Trapani
      Pages 153-175
    6. Benjamin M. Gaub, Michael H. Berry, Meike Visel, Amy Holt, Ehud Y. Isacoff, John G. Flannery
      Pages 177-189
    7. Wen-Hsuan Wu, Yi-Ting Tsai, Sally Justus, Galaxy Y. Cho, Jesse D. Sengillo, Yu Xu et al.
      Pages 191-205
    8. Vithiyanjali Sothilingam, Regine Mühlfriedel, Naoyuki Tanimoto, Mathias W. Seeliger
      Pages 207-213
    9. Regine Mühlfriedel, Marina Garcia Garrido, Christine Wallrapp, Mathias W. Seeliger
      Pages 215-223
    10. Timothy P. Day, Leah C. Byrne, John G. Flannery, David V. Schaffer
      Pages 239-249
    11. Daniyar Dauletbekov, K. Ulrich Bartz-Schmidt, M. Dominik Fischer
      Pages 251-257
  4. Clinical Protocols and Retinal Gene Therapy Vector Testing on Human Retina

    1. Front Matter
      Pages 259-259
    2. Peter M. Quinn, Thilo M. Buck, Charlotte Ohonin, Harald M. M. Mikkers, Jan Wijnholds
      Pages 261-273
    3. Thilo M. Buck, Lucie P. Pellissier, Rogier M. Vos, Elon H. C. van Dijk, Camiel J. F. Boon, Jan Wijnholds
      Pages 275-288
    4. Harry O. Orlans, Thomas L. Edwards, Samantha R. De Silva, Maria I. Patrício, Robert E. MacLaren
      Pages 289-303
    5. Mathieu Gauvin, Allison L. Dorfman, Pierre Lachapelle
      Pages 313-325
    6. Mays Talib, Gislin Dagnelie, Camiel J. F. Boon
      Pages 327-338
    7. Mays Talib, Jasleen K. Jolly, Camiel J. F. Boon
      Pages 339-349
    8. Thomas Theelen, Michel M. Teussink
      Pages 351-358
    9. Alun R. Barnard, Anna N. Rudenko, Robert E. MacLaren
      Pages 359-371
  5. Back Matter
    Pages 373-380

About this book


This detailed volume describes a spectrum of methods and protocols that can be used for the bench-to-bedside development and evaluation of retinal gene therapy. Methods for the successful delivery of these gene therapy vector systems to the retina are examined, as well as assays to test the efficacy in vitro in cell cultures, for gene augmentation and gene editing in vivo on rodents, pigs, and monkey retinas, and on human retinal explants as well as in human clinical studies. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and tips on troubleshooting and avoiding known pitfalls. 

Authoritative and practical, Retinal Gene Therapy: Methods and Protocols provides a wide range of readers from students to research experts with vital information on ocular gene therapy vector technology, in vitro and in vivo biological assays, and clinical protocols, to promote further studies for the benefit of children and adults with inherited retinal disease.


Gene therapy vector Retinal cell types Gene augmentation Cell cultures Gene silencing Vector production AAVs

Editors and affiliations

  • Camiel J.F. Boon
    • 1
  • Jan Wijnholds
    • 2
  1. 1.Department of OphthalmologyLeiden University Medical Center (LUMC)LeidenThe Netherlands
  2. 2.Department of OphthalmologyLeiden University Medical Center (LUMC)LeidenThe Netherlands

Bibliographic information

  • DOI
  • Copyright Information Springer Science+Business Media LLC 2018
  • Publisher Name Humana Press, New York, NY
  • eBook Packages Springer Protocols
  • Print ISBN 978-1-4939-7521-1
  • Online ISBN 978-1-4939-7522-8
  • Series Print ISSN 1064-3745
  • Series Online ISSN 1940-6029
  • Buy this book on publisher's site