Viral Vectors for Gene Therapy: Methods and Protocols

Front Matter ....Pages i-xiv
Front Matter ....Pages 1-1
 Basic Concepts in Viral Vector-Mediated Gene Therapy  (Matthew J. Benskey, Ivette M. Sandoval, Kathryn Miller, Rhyomi L. Sellnow, Aysegul Gezer, Nathan C. Kuhn et al.)....Pages 3-26
Front Matter ....Pages 27-27
 Design and Assembly of CRISPR/Cas9 Lentiviral and rAAV Vectors for Targeted Genome Editing  (Ivette M. Sandoval, Timothy J. Collier, Fredric P. Manfredsson)....Pages 29-45
 Design, Construction, and Application of Transcription Activation-Like Effectors  (Peter Deng, Sakereh Carter, Kyle Fink)....Pages 47-58
 Practical Considerations for the Use of DREADD and Other Chemogenetic Receptors to Regulate Neuronal Activity in the Mammalian Brain  (Patrick Aldrin-Kirk, Tomas Björklund)....Pages 59-87
Front Matter ....Pages 89-89
 AAV Production Using Baculovirus Expression Vector System  (Quentin Sandro, Karima Relizani, Rachid Benchaouir)....Pages 91-99
 Multimodal Production of Adeno-Associated Virus  (Ivette M. Sandoval, Nathan M. Kuhn, Fredric P. Manfredsson)....Pages 101-124
 Generation of High-Titer Pseudotyped Lentiviral Vectors  (Shuang Hu, Mingjie Li, Ramesh Akkina)....Pages 125-134
 A Scalable Lentiviral Vector Production and Purification Method Using Mustang Q Chromatography and Tangential Flow Filtration  (Stuart Tinch, Kathy Szczur, William Swaney, Lilith Reeves, Scott R. Witting)....Pages 135-153
 Current Use of Adenovirus Vectors and Their Production Methods  (Ekramy E. Sayedahmed, Rashmi Kumari, Suresh K. Mittal)....Pages 155-175
 Construction of Oncolytic Herpes Simplex Virus with Therapeutic Genes of Interest  (Andranik Kahramanian, Toshihiko Kuroda, Hiroaki Wakimoto)....Pages 177-188
 Poxviruses as Gene Therapy Vectors: Generating Poxviral Vectors Expressing Therapeutic Transgenes  (Steven J. Conrad, Jia Liu)....Pages 189-209
Front Matter ....Pages 211-211
 AAV-Mediated Gene Delivery to the Mouse Liver  (Sharon C. Cunningham, Ian E. Alexander)....Pages 213-219
 Surgical Methods for Inner Ear Gene Delivery in Neonatal Mouse  (Kevin Isgrig, Wade W. Chien)....Pages 221-226
 Gene Transfer to Mouse Kidney In Vivo  (C. J. Rocca, S. Cherqui)....Pages 227-234
 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, Cristhian J. Ildefonso)....Pages 235-258
 Localized Intra-Arterial Gene Delivery Using AAV  (Koji Hosaka, Fredric P. Manfredsson, Brian L. Hoh)....Pages 259-265
 Stable Genetic Modification of Mesenchymal Stromal Cells Using Lentiviral Vectors  (Francisco Martín, María Tristán-Manzano, Noelia Maldonado-Pérez, Sabina Sánchez-Hernández, Karim Benabdellah, Marién Cobo)....Pages 267-280
 Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs  (Lakmini P. Wasala, Chady H. Hakim, Yongping Yue, N. Nora Yang, Dongsheng Duan)....Pages 281-294
 Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons  (Florie Borel, Eric Adams, Christian Mueller)....Pages 295-303
 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, Carolyn A. Fairbanks)....Pages 305-312
 Cerebellomedullary Cistern Injection of Viral Vectors in Nonhuman Primates  (Lluis Samaranch, Kousaku Ohno, Waldy San Sebastian, Krystof Bankiewicz)....Pages 313-324
Back Matter ....Pages 325-328