This is the PDF eBook version for Muscle Gene Therapy 2nd Edition by Dongsheng Duan, Jerry R. Mendell
Table of Contents
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.- 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.- 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.