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Muscle disease represents an important health threat to the general population. 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. 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.
List of contents
Animal Models for Inherited Muscle Diseases.- In Utero Muscle Gene Transfer.- Gene Therapy for the Respiratory Muscles.- Muscular Dystrophy Gene Therapy in Small Animal Models.- Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy.- Systemic Treatment of Duchenne Muscular Dystrophy by Antisense Oligomer-Induced Exon Skipping.- RNAi Therapy for Dominant Muscular Dystrophies and Other Myopathies.- Combinatorial Gene Therapy Strategies for Treating Muscular Dystrophies.- Duchenne Cardiomyopathy Gene Therapy.- Systemic Gene Delivery for Muscle Gene Therapy.- Modulating Immune Responses in Muscle Gene Therapy.- Delivering Large Therapeutic Genes for Muscle Gene Therapy.- Muscle as a Metabolic Factory for Gene Therapy.- Muscle as a Target for Genetic Vaccine.- Combining Stem Cells and Exon Skipping Strategy to Treat Muscular Dystrophy.- Gene Therapy Clinical Trials for Muscular Dystrophies.
Summary
Muscle disease represents an important health threat to the general population. 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. 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.
