Viral Vectors for Gene Therapy: Methods and Protocols

Preface...
Table of Contents...
Contributing Authors...

Part I Introduction

1. Basic Concepts in Viral Vector-Mediated Gene Therapy
Matthew J. Benskey, Ivette M. Sandoval, Kathryn Miller, Rhyomi L. Sellnow, Aysegul Gezer, Nathan C. Kuhn, Roslyn Vashon, and Fredric P. Manfredsson

Part II Novel Modes of Gene Therapy (Going Beyond Overexpression and Knockdown)

2. Design and Assembly of CRISPR/Cas9 Lentiviral and rAAV Vectors for Targeted Genome Editing
Ivette M. Sandoval, Timothy J. Collier, and Fredric P. Manfredsson

3. Design, Construction, and Application of Transcription Activation-Like Effectors
Peter Deng, Sakereh Carter, and Kyle Fink

4. Practical Considerations for the use of DREADD and Other Chemogenetic Receptors to Regulate Neuronal Activity in the Mammalian Brain
Patrick Aldrin-Kirk and Thomas Björklund

Part III Viral Vectors

5. AAV Production using Baculovirus Expression Vector System
Quentin Sandro, Karima Relizani, and Rachid Benchaouir

6. Multimodal Production of Adeno-Associated Virus
Ivette M. Sandoval, Nathan M. Kuhn, and Fredric P. Manfredsson

7. Generation of High Titer Pseudotyped Lentiviral Particles
Shuang Hu, Mingjie Li, and Ramesh Akkina

8. A Scalable Lentiviral Vector Production and Purification Method using Mustang Q Chromatography and Tangential Flow Filtration
Stuart Tinch, Kathy Szcur, William Swaney, Lilith Reeves, and Scott R. Witting

9. Current Use of Adenovirus Vectors and their Purification Methods
Ekramy E. Sayedahmed, Rashmi Kumari, and Suresh K. Mittal

10. Construction of Oncolytic Herpes Simplex Virus with Therapeutic Genes of Interest
Andranik Kahramanian, Toshihiko Kuroda, and Hiroaki Wakimoto

11. Poxviruses as Gene Therapy Vectors: Generating Poxviral Vectors Expressing Therapeutic Transgenes
Steven Conrad and Jia Liu

Part IV Viral Vector Delivery

12. AAV Mediated Gene Delivery to the Mouse Liver
Sharon C. Cunningham and Ian Alexander

13. Surgical Methods for Inner Ear Gene Delivery in Neonatal Mouse
Kevin Isgrig and Wade Chien

14. Gene Transfer to Mouse Kidney In Vivo
C. J. Rocca and S. Cherqui

15. 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. Bowman, Alfred S. Lewin, and Cristian J. Ildefonso

16. Localized Intra-Arterial Gene Delivery using AAV
Koji Hosaka, Fredric P. Manfredsson, and Brian Hoh

17. Stable Genetic Modification of Mesenchymal Stromal Cells Using Lentiviral Vectors
Francisco Martin, Maria-Tristán-Manzano, Noelia Maldonado, Sabina Sánchez-Hernández, Karim Benabdellah and Marién Cobo

18. Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs
Lakmini P. Wasala, Chady H. Hakim, Yongping Yue, N. Nora Yang, and Donsheng Duan

19. Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons
Florel Borel, Eric Adams, and Christian Mueller

20. Detailed Method for Intrathecal Delivery of Gene Therapeutics by Direct Lumbar Puncture in Mice
Kelsey R. Pfelson, Cristian D. Peterson, Kelley F. Kitto, Lucy Vulchanova, George L. Wilcox, and Carolyn A. Fairbanks

21. Cerebromedullary Cistern Injection of Viral Vectors in Non-Human Primates
Lluis Samaranch, Kousaku Ohno, Waldy San Sebastian, and Krystof Bankiewicz