Muscle gene therapy

"Muscle disease represents an important health threat to the general population. Unlike diseases affecting other tissues/organs, therapeutic options are quite limited for many inherited muscle diseases such as Duchenne muscular dystrophy. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. A variety of new strategies, such as exon-skipping and RNAi, have greatly expanded the scope of muscle gene therapy beyond the traditional gene replacement approach. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy"--Publisher's description

Part 1. Foundations for muscle gene therapy. An overview of muscle biology and physiology for muscle gene therapy -- Molecular basis of muscle disease -- Animal models for muscle disease and muscle gene therapy -- Muscle stem cell biology and implication in gene therapy -- Pluripotent stem cells for gene therapy of hereditary muscle disorders -- MicroRNAs (miRs) in muscle gene therapy -- Immune system regulation of muscle injury and disease -- Design of muscle gene therapy expression cassette -- Chapter 9 Non-viral vector mediated muscle gene therapy -- Viral vectors for muscle gene therapy -- Development of next generation muscle gene therapy AAV vectors -- Histological and biochemical evaluation of muscle gene therapy -- Optical polarization tractography imaging of structural changes in the skeletal and cardiac muscles of the mdx4cv mice -- Biomarkers for muscle disease gene therapy -- Large-scale clinical manufacturing of AAV Vectors for systemic muscle gene therapy -- Genome-editing for muscle gene therapy -- Part 2. Preclinical muscle gene therapy. Considerations on preclinical muscle gene therapy studies -- Gene replacement therapy for Duchenne muscular dystrophy -- Recent a…