Viral vectors for gene therapy : methods and protocols [electronic resource]

  • Manfredsson, Fredric P. , Benskey, Matthew J.
  • Springer
  • 2019
Viral vectors for gene therapy : methods and protocols [electronic resource]


This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. 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.

Table of Content

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
Design and assembly of CRISPR/Cas9 lentiviral and rAAV vectors for targeted genome editing / Ivette M. Sandoval, Timothy J. Collier, and Fredric P. Manfredsson
Design, construction, and application of transcription activation-like effectors / Peter Deng, Sakereh Carter, and Kyle Fink
Practical considerations for the use of DREADD and other chemogenetic receptors to regulate neuronal activity in the mammalian brain / Patrick Aldrin-Kirk and Tomas Bjo¨rklund
AAV production using baculovirus expression vector system / Quentin Sandro, Karima Relizani, and Rachid Benchaouir
Multimodal production of adeno-associated virus / Ivette M. Sandoval, Nathan M. Kuhn, and Fredric P. Manfredsson
Generation of high-titer pseudotyped lentiviral vectors / Shuang Hu, Mingjie Li, and Ramesh Akkina
Scalable lentiviral vector production and purification method using mustang Q chromatography and tangential flow filtration / Stuart Tinch, Kathy Szczur, William Swaney, Lilith Reeves, and Scott R. Witting
Current use of adenovirus vectors and their production methods / Ekramy E. Sayedahmed, Rashmi Kumari, and Suresh K. Mittal
Construction of oncolytic herpes simplex virus with therapeutic genes of interest / Andranik Kahramanian, Toshihiko Kuroda, and Hiroaki Wakimoto
Poxviruses as gene therapy vectors : generating poxviral vectors expressing therapeutic transgenes / Steven J. Conrad and Jia Liu
AAV-mediated gene delivery to the mouse liver / Sharon C. Cunningham and Ian E. Alexander
Surgical methods for inner ear gene delivery in neonatal mouse / Kevin Isgrig and Wade W. Chien
Gene Transfer to Mouse Kidney In Vivo / C. J. Rocca and S. Cherqui
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. Young, Alfred S. Lewin, and Cristhian J. Ildefonso
Localized intra-arterial gene delivery using AAV / Koji Hosaka, Fredric P. Manfredsson, and Brian L. Hoh
Stable genetic modification of mesenchymal stromal cells using lentiviral vectors / Francisco Martı´n, Marı´a Trista´n-Manzano, Noelia Maldonado-Pe´rez, Sabina Sa´nchez-Herna´ ndez, Karim Benabdellah, and Marie´n Cobo
Systemic delivery of adeno-associated viral vectors in mice and dogs / Lakmini P. Wasala, Chady H. Hakim, Yongping Yue, N. Nora Yang, and Dongsheng Duan
Intrathecal delivery of AAV vectors in cynomolgus macaques for CNS gene therapy and gene expression analysis in microdissected motor neurons / Florie Borel, Eric Adams, and Christian Mueller
Detailed method for intrathecal delivery of gene therapeutics by direct lumbar puncture in mice / Kelsey R. Pflepsen, Cristina D. Peterson, Kelley F. Kitto, Lucy Vulchanova, George L. Wilcox, and Carolyn A. Fairbanks
Cerebellomedullary cistern injection of viral vectors in nonhuman primates / Lluis Samaranch, Kousaku Ohno, Waldy San Sebastian, and Krystof Bankiewicz.