Overview
- Editors:
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Dongsheng Duan
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, Dept. of Molecular Microbiology, University of Missouri, Columbia, USA
- 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
- Includes supplementary material: sn.pub/extras
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Table of contents (24 protocols)
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Front Matter
Pages i-xiii
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Basic Methodology Related to Muscle Gene Therapy
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- Charis L. Himeda, Xiaolan Chen, Stephen D. Hauschka
Pages 3-19
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- Takis Athanasopoulos, Helen Foster, Keith Foster, George Dickson
Pages 21-37
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- Glenn Morris, Nguyen thi Man, Caroline A. Sewry
Pages 39-61
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- Jérôme Poupiot, Jérôme Ausseil, Isabelle Richard
Pages 63-74
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- Chady H. Hakim, Dejia Li, Dongsheng Duan
Pages 75-89
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- Brian Bostick, Yongping Yue, Dongsheng Duan
Pages 91-104
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- Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan, Martin K. Childers, Robert W. Grange
Pages 105-123
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New Technology in Muscle Gene Therapy
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Front Matter
Pages 125-125
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- Lin Yang, Juan Li, Xiao Xiao
Pages 127-139
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- Jana L. Phillips, Julia Hegge, Jon A. Wolff, R. Jude Samulski, Aravind Asokan
Pages 141-151
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- Linda J. Popplewell, Ian R. Graham, Alberto Malerba, George Dickson
Pages 153-178
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- Aurélie Goyenvalle, Kay E. Davies
Pages 179-196
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- Zhan-Peng Huang, Ronald L. Neppl Jr., Da-Zhi Wang
Pages 197-210
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- Emmanuel Richard, Gaelle Douillard-Guilloux, Catherine Caillaud
Pages 223-235
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Methods for Muscle Gene Transfer in Large Animal Models
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Front Matter
Pages 237-237
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- Khalil N. Abi-Nader, Anna L. David
Pages 239-256
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- Angela M. Bodles-Brakhop, Ruxandra Draghia-Akli, Kate Broderick, Amir S. Khan
Pages 257-264
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.
Editors and Affiliations
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, Dept. of Molecular Microbiology, University of Missouri, Columbia, USA
Dongsheng Duan
