Methods in Molecular Biology

Muscle Gene Therapy

Methods and Protocols

Editors: Duan, Dongsheng (Ed.)

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  • -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)

Table of contents (24 chapters)
  • Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscles

    Pages 3-19

    Himeda, Charis L. (et al.)

  • Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy

    Pages 21-37

    Athanasopoulos, Takis (et al.)

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

    Pages 39-61

    Morris, Glenn (et al.)

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

    Pages 63-74

    Poupiot, Jérôme (et al.)

  • Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy

    Pages 75-89

    Hakim, Chady H. (et al.)

Buy this book

eBook 118,99 €
price for Spain (gross)
  • ISBN 978-1-61737-982-6
  • Digitally watermarked, DRM-free
  • Included format: EPUB, PDF
  • ebooks can be used on all reading devices
  • Immediate eBook download after purchase
Hardcover 150,79 €
price for Spain (gross)
  • ISBN 978-1-61737-981-9
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
  • The final prices may differ from the prices shown due to specifics of VAT rules
Softcover 150,79 €
price for Spain (gross)
  • ISBN 978-1-4939-6182-5
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
  • The final prices may differ from the prices shown due to specifics of VAT rules
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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