Adeno-Associated Virus: Methods and Protocols

ISBN-13: 9781617793691 / Angielski / Twarda / 2011 / 462 str.

Richard O. Snyder;Philippe Moullier

Opis

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 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.

Spis treści

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

            Jürgen 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. Müller, 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 Büning, 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, Véronique 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