Viral vectors for gene therapy : Methods and protocols

著者

• Manfredsson, Fredric P.
• Benskey, Matthew J.

書誌事項

Viral vectors for gene therapy : Methods and protocols

edited by Fredric P. Manfredsson, Matthew J. Benskey

（Methods in molecular biology / John M. Walker, series editor, 1937）（Springer protocols）

Humana Press, c2019

注記

Includes bibliographical references and index

内容説明・目次

内容説明

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. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.

目次

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 Bjoerklund 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-Tristan-Manzano, Noelia Maldonado, Sabina Sanchez-Hernandez, Karim Benabdellah and Marien 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

「Nielsen BookData」 より

詳細情報

• NII書誌ID(NCID): BB28332926
• ISBN:
  • 9781493990641
• 出版国コード: us
• タイトル言語コード: eng
• 本文言語コード: eng
• 出版地: New York
• ページ数/冊数: xiv, 328 p.
• 大きさ: 26 cm
• 親書誌ID:
  • BA10794673
  • BA88873632