Muscle Gene Therapy: Methods and Protocols by Dongsheng DuanMuscle Gene Therapy: Methods and Protocols by Dongsheng Duan

Muscle Gene Therapy: Methods and Protocols

EditorDongsheng Duan

Hardcover | January 10, 2011

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Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body. In Muscle Gene Therapy: Methods and Protocols, expert researchers in the field present a collection of techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent rodent models and vitally important larger animal models. Divided into three sections, this volume examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle gene therapy technology such as adeno-associated viral vector (AAV), oligonucleotide-mediated exon-skipping, and novel RNA-based strategies, and step-by-step guidance on muscle gene delivery in swine, ovine, canine, and non-human primates. Written in the highly successful Methods in Molecular BiologyT series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, detailed, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls.Authoritative and cutting-edge, Muscle Gene Therapy: Methods and Protocols serves as an invaluable resource for graduate students, post-doctoral fellows, and principle investigators pursuing the crucial advancement of muscle disease gene therapy in the hope of someday curing these debilitating disorders.
Title:Muscle Gene Therapy: Methods and ProtocolsFormat:HardcoverDimensions:395 pagesPublished:January 10, 2011Publisher:Humana PressLanguage:English

The following ISBNs are associated with this title:

ISBN - 10:1617379816

ISBN - 13:9781617379819

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Table of Contents

Part I: Basic Methodology Related to Muscle Gene Therapy1. Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscle Charis L. Himeda, Xiaolan Chen, and Stephen D. Hauschka2. Codon Optimization of the Micro-Dystrophin Gene for Duchene Muscular Dystrophy Gene Therapy Takis Athanasopoulos, Helen Foster, Keith Foster, and George Dickson3. Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal Antibodies Glenn Morris, Nguyen thi Man, and Caroline A. Sewry4. Methods for Non-Invasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter Gene Jérôme Poupiot, Jérôme Ausseil, and Isabelle Richard5. Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy Chady H. Hakim, Dejia Li, and Dongsheng Duan6. Phenotyping Cardiac Gene Therapy in Mice Brian Bostick, Yongping Yue, and Dongsheng Duan7. Golden Retriever Muscular Dystrophy (GRMD): Developing and Maintaining a Colony and Physiological Functional Measurements Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan, Martin K. Childers, and Robert W. GrangePart II: New Technology in Muscle Gene Therapy8. Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy Lin Yang, Juan Li, and Xiao Xiao9. Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV Vectors Jana L. Phillips, Julia Hegge, Jon A. Wolff, R. Jude Samulski, and Aravind Asokan10. Bioinformatic and Functional Optimization of Antisense Phosphorodiamidate Morpholino Oligomers (PMOs) for Therapeutic Modulation of RNA Splicing in Muscle Linda J. Popplewell, Ian R. Graham, Alberto Malerba, and George Dickson11. Engineering Exon-Skipping Vectors Expressing U7snRNA Constructs for Duchenne Muscular Dystrophy Gene Therapy Aurélie Goyenvalle and Kay E. Davies12. Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene Therapy Zhan-Peng Huang, Ronald L. Neppl, Jr., and Da-Zhi Wang13. Molecular Imaging of RNA Interference Therapy Targeting PHD2 for Treatment of Myocardial Ischemia Mei Huang and Joseph C. Wu14. Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target Validation Emmanuel Richard, Gaelle Douillard-Guilloux, and Catherine CaillaudPart III: Methods for Muscle Gene Transfer in Large Animal Models15. Fetal Muscle Gene Therapy/Gene Delivery in Large Animals Khalil N. Abi-Nader and Anna L. David16. Electroporation of Plasmid DNA to Swine Muscle Angela M. Bodles-Brakhop, Ruxandra Draghia-Akli, Kate Broderick, and Amir S. Khan17. Local Gene Delivery and Methods to Control Immune Responses in Muscles of Normal and Dystrophic Dogs Zejing Wang, Stephen J Tapscott, and Rainer Storb18. Gene Transfer to Muscle from the Isolated Regional Circulation Mihail Petrov, Alock Malik, Andrew Mead, Charles Bridges, and Hansell Stedman19. AAV-Mediated Gene Therapy to the Isolated Limb in Rhesus Macaques Louise R. Rodino-Klapac, Chrystal L. Montgomery, Jerry R. Mendell, and Louis G. Chicoine20. Antisense Oligo-Mediated Multiple Exon-Skipping in a Dog Model of Duchenne Muscular Dystrophy Toshifumi Yokota, Eric Hoffman, and Shin'ichi Takeda21. Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9 Yongping Yue, Jin-Hong Shin, and Dongsheng Duan22. A Translatable, Closed Recirculation System for AAV6 Vector-Mediated Myocardial Gene Delivery in the Large Animal JaBaris D. Swain, Michael G. Katz, Jennifer D. White, Danielle M. Thesier, Armen Henderson, Hansell Stedman, and Charles R. Bridges23. Method of Gene Delivery in Large Animal Models of Cardiovascular Diseases Yoshiaki Kawase, Dennis Ladage, and Roger J. Hajjar24. Percutaneous Transendocardial Delivery of Self-Complementary Adeno-Associated Virus 6 in the Canine Lawrence T. Bish, Meg M. Sleeper, and H. Lee Sweeney