Muscle Gene Therapy

1. Front Matter

   Pages i-xiii

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2. Basic Methodology Related to Muscle Gene Therapy

   1. Front Matter

      Pages 1-1

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   2. Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscles

      • Charis L. Himeda, Xiaolan Chen, Stephen D. Hauschka

      Pages 3-19

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   3. Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy

      • Takis Athanasopoulos, Helen Foster, Keith Foster, George Dickson

      Pages 21-37

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   4. Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal Antibodies

      • Glenn Morris, Nguyen thi Man, Caroline A. Sewry

      Pages 39-61

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   5. Methods for Noninvasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter Gene

      • Jérôme Poupiot, Jérôme Ausseil, Isabelle Richard

      Pages 63-74

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   6. Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy

      • Chady H. Hakim, Dejia Li, Dongsheng Duan

      Pages 75-89

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   7. Phenotyping Cardiac Gene Therapy in Mice

      • Brian Bostick, Yongping Yue, Dongsheng Duan

      Pages 91-104

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   8. 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, Robert W. Grange

      Pages 105-123

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3. New Technology in Muscle Gene Therapy

   1. Front Matter

      Pages 125-125

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   2. Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy

      • Lin Yang, Juan Li, Xiao Xiao

      Pages 127-139

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   3. Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV Vectors

      • Jana L. Phillips, Julia Hegge, Jon A. Wolff, R. Jude Samulski, Aravind Asokan

      Pages 141-151

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   4. 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, George Dickson

      Pages 153-178

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   5. Engineering Exon-Skipping Vectors Expressing U7 snRNA Constructs for Duchenne Muscular Dystrophy Gene Therapy

      • Aurélie Goyenvalle, Kay E. Davies

      Pages 179-196

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   6. Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene Therapy

      • Zhan-Peng Huang, Ronald L. Neppl Jr., Da-Zhi Wang

      Pages 197-210

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   7. Molecular Imaging of RNA Interference Therapy Targeting PHD2 for Treatment of Myocardial Ischemia

      • Mei Huang, Joseph C. Wu

      Pages 211-221

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   8. Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target Validation

      • Emmanuel Richard, Gaelle Douillard-Guilloux, Catherine Caillaud

      Pages 223-235

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4. Methods for Muscle Gene Transfer in Large Animal Models

   1. Front Matter

      Pages 237-237

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   2. Fetal Muscle Gene Therapy/Gene Delivery in Large Animals

      • Khalil N. Abi-Nader, Anna L. David

      Pages 239-256

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   3. Electroporation of Plasmid DNA to Swine Muscle

      • Angela M. Bodles-Brakhop, Ruxandra Draghia-Akli, Kate Broderick, Amir S. Khan

      Pages 257-264

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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 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.

• AAV
• Adeno-associated viral vectors
• Delivery systems
• Exon-skipping
• Gene expression cassette
• Gene mutations
• Large animal models
• Muscular degeneration
• gene therapy

• , Dept. of Molecular Microbiology, University of Missouri, Columbia, USA

  Dongsheng Duan

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