Synopses & Reviews
Synopsis
1. Adeno-Associated Virus Biology
Matthew D. Weitzman and R. Michael Linden
2. Design and Construction of Functional AAV Vectors
John T. Gray and Serge Zolotukhin
3. AAV Capsid Structure and Cell Interactions
J rgen Kleinschmidt and Mavis Agbandje-McKenna
4. Exploiting Natural Diversity of AAV for the Design of Vectors With Novel Properties
Guangping Gao, Li Zhong, and Olivier Danos
5. Gene Therapy in Skeletal Muscle Mediated by Adeno-Associated Virus (AAV) Vectors
Chunping Qiao, J. George Dickson, Juan Li, Taeyoung Koo, and Xiao Xiao
6. AAV-Mediated Liver-Directed Gene Therapy
Mark S. Sands
7. Recombinant AAV Delivery to the Central Nervous System
Olivier Bockstael, Kevin Foust, Brian Kaspar, and Liliane Tenenbaum
8. AAV Mediated Gene Therapy for Retinal Degenerative Diseases
Knut Stieger, Therese Cronin, Jean Bennett, and Fabienne Rolling
9. Adeno-Associated Virus Vector Delivery to the Heart
Lawrence T. Bish, H. Lee Sweeney, Oliver J. M ller, and Raffi Bekeredjian
10. Evaluation of the Fate of rAAV Genomes Following in vivo Administration
K. Reed Clark and Magalie Penaud-Budloo
11. Measuring Immune Responses to Recombinant AAV Gene Transfer
Ashley T. Martino, Ignacio Anegon, Oumeya Adjali, and Roland W. Herzog
12. Modification and Labeling of AAV Vector Particles
Hildegard B ning, Chelsea M. Bolyard, Michael Hallek, and Jeffrey S. Bartlett
13. AAV-mediated Gene Targeting
Daniel G. Miller
14. Preclinical Study Design for rAAV
Terence R. Flotte, Thomas J. Conlon, and Christian Mueller
15. Biodistribution and Shedding of AAV Vectors
Caroline le Guiner, Philippe Moullier, and Valder R. Arruda
16. Production and Purification of Recombinant Adeno-Associated Vectors
Lijun Wang, V ronique Blouin, Nicole Brument, Mahajoub Bello-Roufai, and Achille Francois
17. rAAV Vector Product Characterization and Stability Studies
Richard O. Snyder, Muriel Audit, and Joyce D. Francis
18. rAAV Human Trial Experience
Katherine A. High and Patrick Aubourg
Synopsis
Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. The complexity of gene transfer agents in the context of their clinical use requires investigators from a wide variety of backgrounds to have an understanding - or at least an appreciation of - the regulatory environment and constraints that affect vector design, manufacturing, pre-clinical testing, and clinical use, with an emphasis on patient protection. In Adeno-Associated Virus: Methods and Protocols, experts from the United States and Europe have contributed current knowledge of this multi-dimensional field relating to the biology of AAV, rAAV vector design, vector manufacturing and product testing, performance of rAAV vectors in major organs, rAAV-related immunological issues, design of animal and clinical studies, and clinical experience. Written in the successful Methods in Molecular Biology(TM) series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and notes on troubleshooting and avoiding known pitfalls.
Authoritative and accessible, Adeno-Associated Virus: Methods and Protocols provides a complete and comprehensive understanding of this multi-disciplinary and rapidly progressing field.