MPG.eBooks - Table of Contents: Muscle Gene Therapy

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Muscle Gene Therapy

About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just...

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Bibliographic Details
Other Authors:	Duan, Dongsheng (Editor), Mendell, Jerry R. (Editor)
Format:	eBook
Language:	English
Published:	Cham Springer International Publishing 2019, 2019
Edition:	2nd ed. 2019
Subjects:	Developmental Biology Virology Immunology Human Genetics
Online Access:	https://doi.org/10.1007/978-3-030-03095-7?nosfx=y
Collection:	Springer eBooks 2005- - Collection details see MPG.ReNa

Table of Contents:

Chapter 38. Design of clinical trials for gene therapy in muscular dystrophy
Chapter 39. Path to clinical trials: trial design, development of the clinical product and safety concerns in the implementation of clinical trials
Chapter 40. Muscle MRI as an endpoint in clinical trials
Chapter 41. Gene therapy clinical trials for Duchenne and limb girdle muscular dystrophies lessons learned
Chapter 42. Duchenne muscular dystrophy exon-skipping trials
Chapter 43. What we have learned from 10 years of DMD exon skipping trials
Chapter 44. Clinical gene therapy trials for Pompe disease
Chapter 45. Muscle-directed gene therapy for alpha-1 antitrypsin deficiency
Chapter 26. Muscle cell membrane repair and therapeutic implications
Chapter 27. Treatment of muscular dystrophy-dystroglycanopathy: unlocking the potential of genetic engineering
Chapter 28. RNAi therapy for dominant muscular dystrophies and other myopathies
Chapter 29. Gene therapy for facioscapulohumeral muscular dystrophy (FSHD)
Chapter 30. Gene therapy and gene editing for myotonic dystrophy
Chapter 31. Gene therapy for oculopharyngeal muscular dystrophy
Chapter 32. Gene therapy for X-linked myotubular myopathy
Chapter 33. Pre-clinical gene therapy studies for metabolic myopathy
Chapter 34 Elimination of mutant mitochondrial DNA in mitochondrial myopathies using gene editing enzymes
Chapter 35. Gene therapy for Charcot-Marie-Tooth inherited neuropathy
Chapter 36. Muscle - a potent target in vaccination
Part 3 Clinical muscle gene therapy
Chapter 37. Patient and family perspective on muscle gene therapy
Part 1 Foundations for muscle gene therapy
Chapter 1. An overview of muscle biology and physiology for muscle gene therapy
Chapter 2. Molecular basis of muscle disease
Chapter 3. Animal models for muscle disease and muscle gene therapy
Chapter 4. Muscle stem cell biology and implication in gene therapy
Chapter 5. Pluripotent stem cells for gene therapy of hereditary muscle disorders
Chapter 6. MicroRNAs (miRs) in muscle gene therapy
Chapter 7. Immune system regulation of muscle injury and disease
Chapter 8. Design of muscle gene therapy expression cassette
Chapter 9 Non-viral vector mediated muscle gene therapy
Chapter 10. Viral vectors for muscle gene therapy
Chapter 11. Development of next generation muscle gene therapy AAV vectors
Chapter 12. Histological and biochemical evaluation of muscle gene therapy
Chapter 13. Optical polarization tractography imaging of structural changes in the skeletal and cardiac muscles of the mdx4cv mice
Chapter 14. Biomarkers for muscle disease gene therapy
Chapter 15. Large-scale clinical manufacturing of AAV Vectors for systemic muscle gene therapy
Chapter 16. Genome-editing for muscle gene therapy
Part 2 Preclinical muscle gene therapy
Chapter 17. Considerations on preclinical muscle gene therapy studies
Chapter 18. Gene replacement therapy for Duchenne muscular dystrophy
Chapter 19. Recent advances in AON-mediated exon-skipping therapy for Duchenne muscular dystrophy
Chapter 20. AAV-mediated exon-skipping therapy for Duchenne muscular dystrophy
Chapter 21. Alternate translational initiation of dystrophin: a novel therapeutic approach
Chapter 22. Genome editing for Duchenne muscular dystrophy
Chapter 23. Sarcolipin knockdown therapy for Duchenne muscular dystrophy
Chapter 24. Gene therapy for central nervous system in Duchenne muscular dystrophy
Chapter 25. Therapeutic approach for dysferlinopathy in animal models

Muscle Gene Therapy

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