Muscle Gene Therapy: Methods and Protocols

ISBN-13: 9781617379819 / Angielski / Twarda / 2011 / 398 str.

Dongsheng Duan

Opis

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 Biology 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.

Spis treści

Part I: Basic Methodology Related to Muscle Gene Therapy 1. Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscle Charis L. Himeda, Xiaolan Chen, and Stephen D. Hauschka 2. Codon Optimization of the Micro-Dystrophin Gene for Duchene Muscular Dystrophy Gene Therapy Takis Athanasopoulos, Helen Foster, Keith Foster, and George Dickson 3. Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal Antibodies Glenn Morris, Nguyen thi Man, and Caroline A. Sewry 4. Methods for Non-Invasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter Gene Jérôme Poupiot, Jérôme Ausseil, and Isabelle Richard 5. Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy Chady H. Hakim, Dejia Li, and Dongsheng Duan 6. Phenotyping Cardiac Gene Therapy in Mice Brian Bostick, Yongping Yue, and Dongsheng Duan 7. 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. Grange Part II: New Technology in Muscle Gene Therapy 8. Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy Lin Yang, Juan Li, and Xiao Xiao 9. Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV Vectors Jana L. Phillips, Julia Hegge, Jon A. Wolff, R. Jude Samulski, and Aravind Asokan 10. 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 Dickson 11. Engineering Exon-Skipping Vectors Expressing U7snRNA Constructs for Duchenne Muscular Dystrophy Gene Therapy Aurélie Goyenvalle and Kay E. Davies 12. Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene Therapy Zhan-Peng Huang, Ronald L. Neppl, Jr., and Da-Zhi Wang 13. Molecular Imaging of RNA Interference Therapy Targeting PHD2 for Treatment of Myocardial Ischemia Mei Huang and Joseph C. Wu 14. Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target Validation Emmanuel Richard, Gaelle Douillard-Guilloux, and Catherine Caillaud Part III: Methods for Muscle Gene Transfer in Large Animal Models 15. Fetal Muscle Gene Therapy/Gene Delivery in Large Animals Khalil N. Abi-Nader and Anna L. David 16. Electroporation of Plasmid DNA to Swine Muscle Angela M. Bodles-Brakhop, Ruxandra Draghia-Akli, Kate Broderick, and Amir S. Khan 17. Local Gene Delivery and Methods to Control Immune Responses in Muscles of Normal and Dystrophic Dogs Zejing Wang, Stephen J Tapscott, and Rainer Storb 18. Gene Transfer to Muscle from the Isolated Regional Circulation Mihail Petrov, Alock Malik, Andrew Mead, Charles Bridges, and Hansell Stedman 19. AAV-Mediated Gene Therapy to the Isolated Limb in Rhesus Macaques Louise R. Rodino-Klapac, Chrystal L. Montgomery, Jerry R. Mendell, and Louis G. Chicoine 20. Antisense Oligo-Mediated Multiple Exon-Skipping in a Dog Model of Duchenne Muscular Dystrophy Toshifumi Yokota, Eric Hoffman, and Shin’ichi Takeda 21. Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9 Yongping Yue, Jin-Hong Shin, and Dongsheng Duan 22. 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. Bridges 23. Method of Gene Delivery in Large Animal Models of Cardiovascular Diseases Yoshiaki Kawase, Dennis Ladage, and Roger J. Hajjar 24. Percutaneous Transendocardial Delivery of Self-Complementary Adeno-Associated Virus 6 in the Canine Lawrence T. Bish, Meg M. Sleeper, and H. Lee Sweeney