BookDongsheng Duan, Jerry R. Mendell, editors.
Summary: An updated overview of muscle gene therapy is provided in this comprehensive reference. The test highlights important gene therapy applications, in addition to the potential healing capabilities in complex genetic disorders.
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