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Adeno-Associated Virus
Methods and Protocols
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Main description:

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.


Contents:

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

Jurgen 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. Muller, 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 Buning, 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, Veronique 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


PRODUCT DETAILS

ISBN-13: 9781617793691
Publisher: Springer (Humana Press Inc.)
Publication date: October, 2011
Pages: 475
Weight: 1085g
Availability: Available
Subcategories: Genetics
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