- -Presents a comprehensive collection of state-of-the-art muscle gene therapy protocols from leaders in the field
- -Focuses on gene delivery in less-covered large animal models, such as swine, ovine, canine, and non-human primates
- -Contains tips from the experts highlighting key implementation advice and strategies
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- About this book
-
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.
- Table of contents (24 chapters)
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Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscles
Pages 3-19
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Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy
Pages 21-37
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Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal Antibodies
Pages 39-61
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Methods for Noninvasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter Gene
Pages 63-74
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Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy
Pages 75-89
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Table of contents (24 chapters)
- Download Preface 1 PDF (181.7 KB)
- Download Sample pages 1 PDF (2 MB)
- Download Table of contents PDF (1.4 MB)
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Bibliographic Information
- Bibliographic Information
-
- Book Title
- Muscle Gene Therapy
- Book Subtitle
- Methods and Protocols
- Editors
-
- Dongsheng Duan
- Series Title
- Methods in Molecular Biology
- Series Volume
- 709
- Copyright
- 2011
- Publisher
- Humana Press
- Copyright Holder
- Springer Science+Business Media, LLC
- eBook ISBN
- 978-1-61737-982-6
- DOI
- 10.1007/978-1-61737-982-6
- Hardcover ISBN
- 978-1-61737-981-9
- Softcover ISBN
- 978-1-4939-6182-5
- Series ISSN
- 1064-3745
- Edition Number
- 1
- Number of Pages
- XIII, 382
- Topics
