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
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.
Part I: Basic Methodology Related to Muscle Gene Therapy1. Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac MuscleCharis L. Himeda, Xiaolan Chen, and Stephen D. Hauschka2. Codon Optimization of the Micro-Dystrophin Gene for Duchene Muscular Dystrophy Gene TherapyTakis Athanasopoulos, Helen Foster, Keith Foster, and George Dickson3. Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal AntibodiesGlenn Morris, Nguyen thi Man, and Caroline A. Sewry4. Methods for Non-Invasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter GeneJérôme Poupiot, Jérôme Ausseil, and Isabelle Richard5. Monitoring Murine Skeletal Muscle Function for Muscle Gene TherapyChady H. Hakim, Dejia Li, and Dongsheng Duan6. Phenotyping Cardiac Gene Therapy in MiceBrian Bostick, Yongping Yue, and Dongsheng Duan7. Golden Retriever Muscular Dystrophy (GRMD): Developing and Maintaining a Colony and Physiological Functional MeasurementsJoe N. Kornegay, Janet R. Bogan, Daniel J. Bogan, Martin K. Childers, and Robert W. GrangePart II: New Technology in Muscle Gene Therapy8. Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene TherapyLin Yang, Juan Li, and Xiao Xiao9. Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV VectorsJana L. Phillips, Julia Hegge, Jon A. Wolff, R. Jude Samulski, and Aravind Asokan10. Bioinformatic and Functional Optimization of Antisense Phosphorodiamidate Morpholino Oligomers (PMOs) for Therapeutic Modulation of RNA Splicing in MuscleLinda J. Popplewell, Ian R. Graham, Alberto Malerba, and George Dickson11. Engineering Exon-Skipping Vectors Expressing U7snRNA Constructs for Duchenne Muscular Dystrophy Gene TherapyAurélie Goyenvalle and Kay E. Davies12. Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene TherapyZhan-Peng Huang, Ronald L. Neppl, Jr., and Da-Zhi Wang13. Molecular Imaging of RNA Interference Therapy Targeting PHD2 for Treatment of Myocardial IschemiaMei Huang and Joseph C. Wu14. Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target ValidationEmmanuel Richard, Gaelle Douillard-Guilloux, and Catherine CaillaudPart III: Methods for Muscle Gene Transfer in Large Animal Models15. Fetal Muscle Gene Therapy/Gene Delivery in Large AnimalsKhalil N. Abi-Nader and Anna L. David16. Electroporation of Plasmid DNA to Swine MuscleAngela M. Bodles-Brakhop, Ruxandra Draghia-Akli, Kate Broderick, and Amir S. Khan17. Local Gene Delivery and Methods to Control Immune Responses in Muscles of Normal and Dystrophic DogsZejing Wang, Stephen J Tapscott, and Rainer Storb18. Gene Transfer to Muscle from the Isolated Regional CirculationMihail Petrov, Alock Malik, Andrew Mead, Charles Bridges, and Hansell Stedman19. AAV-Mediated Gene Therapy to the Isolated Limb in Rhesus MacaquesLouise R. Rodino-Klapac, Chrystal L. Montgomery, Jerry R. Mendell, and Louis G. Chicoine20. Antisense Oligo-Mediated Multiple Exon-Skipping in a Dog Model of Duchenne Muscular DystrophyToshifumi Yokota, Eric Hoffman, and Shin´ichi Takeda21. Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9Yongping Yue, Jin-Hong Shin, and Dongsheng Duan22. A Translatable, Closed Recirculation System for AAV6 Vector-Mediated Myocardial Gene Delivery in the Large AnimalJaBaris D. Swain, Michael G. Katz, Jennifer D. White, Danielle M. Thesier, Armen Henderson, Hansell Stedman, and Charles R. Bridges23. Method of Gene Delivery in Large Animal Models of Cardiovascular DiseasesYoshiaki Kawase, Dennis Ladage, and Roger J. Hajjar24. Percutaneous Transendocardial Delivery of Self-Complementary Adeno-Associated Virus 6 in the CanineLawrence T. Bish, Meg M. Sleeper, and H. Lee Sweeney
Written in the popular Methods in Molecular Biology(TM) series format, this book presents 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.
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(TM) 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.
Design and Testing of Regulatory Cassettes for Optimal Activity in Skeletal and Cardiac Muscle.- Codon Optimization of the Micro-Dystrophin Gene for Duchene Muscular Dystrophy Gene Therapy.- Monitoring Duchenne Muscular Dystrophy Gene Therapy with Epitope-Specific Monoclonal Antibodies.- Methods for Non-Invasive Monitoring of Muscle Fiber Survival with an AAV Vector Encoding the mSEAP Reporter Gene.- Monitoring Murine Skeletal Muscle Function for Muscle Gene Therapy.- Phenotyping Cardiac Gene Therapy in Mice.- Golden Retriever Muscular Dystrophy (GRMD): Developing and Maintaining a Colony and Physiological Functional Measurements.- Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy.- Systemic Gene Transfer to Skeletal Muscle Using Reengineered AAV Vectors.- Bioinformatic and Functional Optimization of Antisense Phosphorodiamidate Morpholino Oligomers (PMOs) for Therapeutic Modulation of RNA Splicing in Muscle.- Engineering Exon-Skipping Vectors Expressing U7snRNA Constructs for Duchenne Muscular Dystrophy Gene Therapy.- Application of MicroRNA in Cardiac and Skeletal Muscle Disease Gene Therapy.- Molecular Imaging of RNA Interference Therapy Targeting PHD2 for Treatment of Myocardial Ischemia.- Lentiviral Vector Delivery of shRNA into Cultured Primary Myogenic Cells: A Tool for Therapeutic Target Validation.- Fetal Muscle Gene Therapy/Gene Delivery in Large Animals.- Electroporation of Plasmid DNA to Swine Muscle.- Local Gene Delivery and Methods to Control Immune Responses in Muscles of Normal and Dystrophic Dogs.- Gene Transfer to Muscle from the Isolated Regional Circulation.- AAV-Mediated Gene Therapy to the Isolated Limb in Rhesus Macaques.- Antisense Oligo-Mediated Multiple Exon-Skipping in a Dog Model of Duchenne Muscular Dystrophy.- Whole Body Skeletal Muscle Transduction in Neonatal Dogs with AAV-9.- A Translatable, Closed Recirculation System for AAV6 Vector-Mediated Myocardial Gene Delivery in the Large Animal.- Method of Gene Delivery in Large Animal Models of Cardiovascular Diseases.- Percutaneous Transendocardial Delivery of Self-Complementary Adeno-Associated Virus 6 in the Canine.
Inhaltsverzeichnis
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
Klappentext
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.nnAuthoritative 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.
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
