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 about to enter its prime time. The progress made since then has been unprecedented. The number of diseases that have been targeted by gene therapy has increased tremendously. The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes). Most importantly, clinical benefits have begun to emerge in human patients. To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients. The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy. It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years. This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community." - Prové de l'editor.

Conté: 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 t…