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Table of Contents
Intro; Dedication; Preface; Disclosures; Contents; Part I: Foundations for Muscle Gene Therapy; Chapter 1: An Overview of Muscle Biology and Physiology for Muscle Gene Therapy; 1.1 Skeletal Muscle; 1.1.1 Skeletal Muscle Structure Overview; 1.1.2 Sarcomere Organization; 1.1.3 Sarcomere Function; 1.1.4 Twitch Contraction; 1.1.5 Tetanic Contraction; 1.1.6 Motor Units; 1.1.7 Fiber Sub-Types; 1.1.8 Modes of Contraction; 1.1.9 Length Tension Relationship; 1.1.10 Lever Action; 1.1.11 Muscular Dystrophy and Skeletal Muscle Contraction; 1.2 Cardiac Muscle; 1.2.1 Cardiac Muscle Structure Overview
1.2.2 Cardiac Muscle Function1.2.3 Muscular Dystrophy and Cardiac Muscle Contraction; References; Chapter 2: Molecular Basis of Muscle Disease; 2.1 Introduction; 2.1.1 Skeletal Muscle Fiber Types and Adaptation; 2.1.2 Regeneration and Satellite Cells; 2.1.3 Primary Muscle Disorders; 2.2 Congenital Myopathies; 2.2.1 Nemaline Myopathies; 2.2.1.1 Nebulin; 2.2.1.2 Kelch Proteins; 2.2.1.3 Leiomodin-3 (LMOD3); 2.2.2 Centronuclear Myopathy; 2.2.3 Core Myopathies; 2.3 Muscular Dystrophies; 2.3.1 Duchenne Muscular Dystrophy; 2.3.2 Limb-Girdle Muscular Dystrophies
2.3.2.1 LGMD Caused by Mutations in Sarcoglycans2.3.2.2 LGMD Caused by Mutations in Sarcolemmal Repair Complex; 2.3.3 Congenital Muscular Dystrophy; 2.3.4 Facioscapulohumeral Muscular Dystrophy (FSHD); 2.3.5 Myotonic Dystrophy; 2.3.6 Emery-Dreifuss Muscular Dystrophy (EDMD); 2.4 Concluding Remarks; References; Chapter 3: Animal Models for Muscle Disease and Muscle Gene Therapy; 3.1 Introduction; 3.2 Dystrophinopathies; 3.3 Limb-Girdle Muscular Dystrophies; 3.3.1 Myotilinopathies; 3.3.2 Laminopathies; 3.3.3 Caveolinopathies; 3.3.4 Calpainopathies; 3.3.5 Dysferlinopathies
3.3.6 Sarcoglycanopathies3.3.7 TRIM32 Mutations; 3.3.8 Fukutin-Related Protein Mutations; 3.4 Summary and Future Direction; References; Chapter 4: Muscle Stem Cell Biology and Implications in Gene Therapy; 4.1 What Is a 'Stem Cell'?; 4.2 What Is a 'Muscle Stem Cell'?; 4.3 Practical Utility of Muscle Stem Cells; 4.4 Stem Cells as Vectors of Genetic Modification; 4.5 Stem Cell Function as a Target of Genetic Therapies; 4.6 Hopes, Aspirations and Hurdles; References; Chapter 5: Pluripotent Stem Cells for Gene Therapy of Hereditary Muscle Disorders; 5.1 Introduction
5.2 Myogenic Adult Stem/Progenitor Cells for Muscle Regeneration5.3 ES and iPS Cells for Muscle Regeneration; 5.4 Genetic Modification of Pluripotent Stem Cells; 5.4.1 Gene Addition; 5.4.2 Gene Editing; 5.5 Concluding Remarks and Future Perspectives; References; Chapter 6: MicroRNAs (miRs) in Muscle Gene Therapy; 6.1 Introduction: miR Biogenesis and Delivery; 6.2 MiRs and Skeletal Myogenesis; 6.3 MiR-Based Therapy for Skeletal Muscle Diseases; 6.4 MiRs and Cardiac Myogenesis; 6.5 MiR-Based Therapy for Cardiac and Smooth Muscle Diseases; 6.6 Conclusion and Remarks; References
1.2.2 Cardiac Muscle Function1.2.3 Muscular Dystrophy and Cardiac Muscle Contraction; References; Chapter 2: Molecular Basis of Muscle Disease; 2.1 Introduction; 2.1.1 Skeletal Muscle Fiber Types and Adaptation; 2.1.2 Regeneration and Satellite Cells; 2.1.3 Primary Muscle Disorders; 2.2 Congenital Myopathies; 2.2.1 Nemaline Myopathies; 2.2.1.1 Nebulin; 2.2.1.2 Kelch Proteins; 2.2.1.3 Leiomodin-3 (LMOD3); 2.2.2 Centronuclear Myopathy; 2.2.3 Core Myopathies; 2.3 Muscular Dystrophies; 2.3.1 Duchenne Muscular Dystrophy; 2.3.2 Limb-Girdle Muscular Dystrophies
2.3.2.1 LGMD Caused by Mutations in Sarcoglycans2.3.2.2 LGMD Caused by Mutations in Sarcolemmal Repair Complex; 2.3.3 Congenital Muscular Dystrophy; 2.3.4 Facioscapulohumeral Muscular Dystrophy (FSHD); 2.3.5 Myotonic Dystrophy; 2.3.6 Emery-Dreifuss Muscular Dystrophy (EDMD); 2.4 Concluding Remarks; References; Chapter 3: Animal Models for Muscle Disease and Muscle Gene Therapy; 3.1 Introduction; 3.2 Dystrophinopathies; 3.3 Limb-Girdle Muscular Dystrophies; 3.3.1 Myotilinopathies; 3.3.2 Laminopathies; 3.3.3 Caveolinopathies; 3.3.4 Calpainopathies; 3.3.5 Dysferlinopathies
3.3.6 Sarcoglycanopathies3.3.7 TRIM32 Mutations; 3.3.8 Fukutin-Related Protein Mutations; 3.4 Summary and Future Direction; References; Chapter 4: Muscle Stem Cell Biology and Implications in Gene Therapy; 4.1 What Is a 'Stem Cell'?; 4.2 What Is a 'Muscle Stem Cell'?; 4.3 Practical Utility of Muscle Stem Cells; 4.4 Stem Cells as Vectors of Genetic Modification; 4.5 Stem Cell Function as a Target of Genetic Therapies; 4.6 Hopes, Aspirations and Hurdles; References; Chapter 5: Pluripotent Stem Cells for Gene Therapy of Hereditary Muscle Disorders; 5.1 Introduction
5.2 Myogenic Adult Stem/Progenitor Cells for Muscle Regeneration5.3 ES and iPS Cells for Muscle Regeneration; 5.4 Genetic Modification of Pluripotent Stem Cells; 5.4.1 Gene Addition; 5.4.2 Gene Editing; 5.5 Concluding Remarks and Future Perspectives; References; Chapter 6: MicroRNAs (miRs) in Muscle Gene Therapy; 6.1 Introduction: miR Biogenesis and Delivery; 6.2 MiRs and Skeletal Myogenesis; 6.3 MiR-Based Therapy for Skeletal Muscle Diseases; 6.4 MiRs and Cardiac Myogenesis; 6.5 MiR-Based Therapy for Cardiac and Smooth Muscle Diseases; 6.6 Conclusion and Remarks; References