Regenerative medicine for degenerative muscle diseases. ([2015])

• Record Type: Book
• Further Information: Note: Martin K. Childers, editor.
• Editors: Childers, Martin K
• Contents: 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. 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. 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. 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. 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.
• Publisher Details: New York : Humana Press
• Publication Date: 2015
• Copyright Date: 2016
• Extent: 1 online resource (xiii, 336 pages), illustrations (some color)
• Subjects: 616.02/7; Life sciences; Regenerative medicine; Muscles -- Diseases; Muscles -- Diseases; Regenerative medicine; Regenerative Medicine; Muscular Diseases; Science -- Biotechnology; Biotechnology; Stem cells; Biotechnology; Science -- Life Sciences -- Cytology; Genetics (non-medical); Electronic books
• Languages: English
• ISBNs: 9781493932283; 1493932284
• Related ISBNs: 9781493932276; 1493932276
• Access Rights: Legal Deposit; Only available on premises controlled by the deposit library and to one user at any one time; The Legal Deposit Libraries (Non-Print Works) Regulations (UK).
• Access Usage: Restricted: Printing from this resource is governed by The Legal Deposit Libraries (Non-Print Works) Regulations (UK) and UK copyright law currently in force.