Adeno-associated virus : methods and protocols / edited by Richard O. Snyder, Philippe Moullier.

Contributor(s): Snyder, Richard O. (Richard Owen) | Moullier, Philippe
Material type: TextTextSeries: Methods in molecular biology (Clifton, N.J.): v. 807.Publisher: Totowa, NJ : Humana Press, ©2011Description: 1 online resource (xiii, 462 pages)Content type: text Media type: computer Carrier type: online resourceISBN: 9781617793707; 1617793701; 1617793698; 9781617793691Subject(s): Gene therapy -- Laboratory manuals | Genetic transformation -- Laboratory manuals | Parvoviruses -- Laboratory manuals | Genetic vectors -- Laboratory manuals | Dependoviruses | Dependovirus | Parvovirinae | Genetic Techniques | Genetic Structures | Publication Formats | Investigative Techniques | Parvoviridae | Genetic Phenomena | DNA Viruses | Viruses | Genetic Vectors | Methods | Gene Transfer Techniques | Dependoviruses | Gene therapy | Genetic transformation | Genetic vectors | Parvoviruses | Medicine | Medicine & Public Health | geneeskunde | volksgezondheid | public health | infectieziekten | infectious diseases | gentherapie | gene therapy | Medicine (General) | Geneeskunde (algemeen)Genre/Form: Laboratory Manual. | Electronic books. | Laboratory manuals. | Laboratory manuals. Additional physical formats: Print version:: Adeno-associated virus.DDC classification: 615.8/95 LOC classification: RB155.8 | .A34 2011Online resources: Click here to access online
Contents:
Adeno-associated virus biology / Matthew D. Weitzman and R. Michael Linden -- Design and construction of functional AAV vectors / John T. Gray and Serge Zolotukhin -- AAV capsid structure and cell interactions / Mavis Agbandje-McKenna and Jürgen Kleinschmidt -- Exploring natural diversity of AAV for the design of vectors with novel properties / Guangping Gao, Li Zhong, and Olivier Danos -- Gene therapy in skeletal muscle mediated by adeno-associated virus vectors / Chunping Qiao [and others] -- AAV mediated liver-directed gene therapy / Mark S. Sands -- Recombinant AAV delivery to the central nervous system / Olivier Bockstael [and others] -- Adeno-associated virus mediated gene therapy for retinal degenerative diseases / Knut Steiger [and others] -- Adeno-associated virus vector delivery to the heart / Lawrence T. Bish [and others] -- Evaluation of the fate of rAAV genomes following in vivo administration / K. Reed Clark and Magalie Penaud-Budloo -- Measuring immune responses to recombinant AAV gene transfer / Ashley T. Martino [and others] -- Modificaton and labeling of AAV vector particles / Hildegard Büning [and others] -- AAV mediated gene targeting / Daniel G. Miller -- Preclinical study design for rAAV / Terence R. Flotte, Thomas J. Conlon, and Christian Mueller -- Biodistribution and shedding of AAV vectors / Caroline Le Guiner, Phillipe Moullier, and Valder R. Arruda -- Production and purification of recombinant adeno-associated vectors / Lijun Wang [and others] -- rAAV vector product characterization and stability studies / Richard O. Snyder, Muriel Audit, and Joyce D. Francis -- rAAV human trial experience / Katherine A. High and Patrick Aubourg.
Summary: 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.
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Includes bibliographical references and index.

Adeno-associated virus biology / Matthew D. Weitzman and R. Michael Linden -- Design and construction of functional AAV vectors / John T. Gray and Serge Zolotukhin -- AAV capsid structure and cell interactions / Mavis Agbandje-McKenna and Jürgen Kleinschmidt -- Exploring natural diversity of AAV for the design of vectors with novel properties / Guangping Gao, Li Zhong, and Olivier Danos -- Gene therapy in skeletal muscle mediated by adeno-associated virus vectors / Chunping Qiao [and others] -- AAV mediated liver-directed gene therapy / Mark S. Sands -- Recombinant AAV delivery to the central nervous system / Olivier Bockstael [and others] -- Adeno-associated virus mediated gene therapy for retinal degenerative diseases / Knut Steiger [and others] -- Adeno-associated virus vector delivery to the heart / Lawrence T. Bish [and others] -- Evaluation of the fate of rAAV genomes following in vivo administration / K. Reed Clark and Magalie Penaud-Budloo -- Measuring immune responses to recombinant AAV gene transfer / Ashley T. Martino [and others] -- Modificaton and labeling of AAV vector particles / Hildegard Büning [and others] -- AAV mediated gene targeting / Daniel G. Miller -- Preclinical study design for rAAV / Terence R. Flotte, Thomas J. Conlon, and Christian Mueller -- Biodistribution and shedding of AAV vectors / Caroline Le Guiner, Phillipe Moullier, and Valder R. Arruda -- Production and purification of recombinant adeno-associated vectors / Lijun Wang [and others] -- rAAV vector product characterization and stability studies / Richard O. Snyder, Muriel Audit, and Joyce D. Francis -- rAAV human trial experience / Katherine A. High and Patrick Aubourg.

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.

English.

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