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BL
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| Record Number
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644762
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| Doc. No
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dltt
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| Title & Author
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Regenerative medicine for degenerative muscle diseases /\ Martin K. Childers, editor
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| Series Statement
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Stem cell biology and regenerative medicine,
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| Page. NO
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1 online resource (xiii, 336 pages) :: illustrations (some color).
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| ISBN
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9781493932283
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: 1493932284
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9781493932276
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1493932276
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| Notes
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Includes index
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| Contents
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Preface; About the Editor; Contents; Chapter 1: Regenerative Medicine Approaches to Degenerative Muscle Diseases; 1.1 Background; 1.1.1 What Is Regenerative Medicine?; 1.1.2 Degenerative Muscle Diseases Are Rare, but Rare Diseases Are Common; 1.2 What Disorders Lead to Chronic Muscle Degeneration?; 1.2.1 Myopathies; 1.2.2 Acquired Myopathies; 1.2.3 Congenital Myopathies; 1.2.4 Muscular Dystrophies; 1.3 Can Human Cells Be Transplanted to Regenerate Damaged Muscles?; 1.3.1 Adult Stem Cells; 1.3.2 Embryonic Stem Cells; 1.3.3 Cellular Reprogramming
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1.4 What Is Gene Therapy and When Will It Be Used in Clinical Practice?1.5 Will Gene Replacement Therapy Be Useful for Inherited Muscle Diseases like Muscular Dystrophy?; 1.6 Can Genes Be Repaired?; 1.7 Can Gene Replacement Therapy Rescue Lethal Muscle Disease? Lessons Learned from Dogs; References; Chapter 2: An Overview of rAAV Vector Product Development for Gene Therapy; 2.1 Vector Design and Lead Identification; 2.2 Product Manufacturing; 2.2.1 Scale; 2.2.2 Reagents and Materials; 2.2.3 Process Design and Development; 2.2.4 Upstream Processing; 2.2.5 Downstream Processing
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2.2.6 Formulation, Fill, Finish, and Product Configuration2.3 Analytical Testing; 2.3.1 Assay Design and Development; 2.3.2 Assay Qualification; 2.3.3 Sampling and Testing; 2.3.4 Product Specifications Setting; 2.3.5 Stability Testing; 2.4 Toxicology Studies; 2.5 Clinical Trials; 2.6 Regulatory; 2.6.1 Expedited Programs; 2.7 Commercial; References; Chapter 3: Gene Discovery in Congenital Myopathy; 3.1 Congenital Myopathies; 3.1.1 Overview; 3.1.2 Nemaline Myopathies; 3.1.2.1 Clinical Features; 3.1.2.2 Pathological Features; 3.1.2.3 Genetics; 3.1.3 Core Myopathies; 3.1.3.1 Clinical Features
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3.1.3.2 Pathological Features3.1.3.3 Genetics; 3.1.4 Centronuclear Myopathies; 3.1.4.1 Clinical Features; 3.1.4.2 Pathological Features; 3.1.4.3 Genetics; 3.1.5 Congenital Fiber-Type Disproportion; 3.1.5.1 Clinical and Pathological Features; 3.1.5.2 Genetics; 3.1.6 Undefined Congenital Myopathies; 3.2 Gene Discovery in Congenital Myopathies; 3.2.1 Diagnostic Challenges; 3.2.1.1 Genetic Heterogeneity; 3.2.1.2 Clinical Heterogeneity; 3.2.1.3 Lack of Specific Biomarkers; 3.2.2 Genetic Approaches; 3.2.2.1 Linkage Mapping and Positional Cloning; 3.2.2.2 Homozygosity Mapping
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3.2.2.3 Candidate Gene Studies3.2.2.4 Next-Generation Sequencing; 3.2.3 Clinical Implications of NGS; 3.2.3.1 NGS Gene Panels; 3.2.3.2 Structural Variation; 3.3 Development of Targeted Therapies; 3.4 Conclusion; References; Chapter 4: Stem Cell Transplantation for Degenerative Muscle Diseases; 4.1 Introduction to Duchenne Muscular Dystrophy; 4.2 Stem Cell Transplantation for the Treatment of DMD; 4.2.1 Satellite Cells/Myoblasts; 4.2.2 Muscle-Derived Stem Cells; 4.2.3 Myoendothelial Cells and Pericytes; 4.2.4 Mesoangioblasts; 4.2.5 CD133+ Cells; 4.2.6 Mesenchymal Stem Cells
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| Subject
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Regenerative medicine.
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| Subject
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Muscles-- Diseases.
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| Dewey Classification
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616.02/7
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| LC Classification
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R857.T55
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| Added Entry
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Childers, Martin K.
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| Added Entry
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Ohio Library and Information Network.
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