Muscle Gene Therapy

ISBN-13: 9781489985248 / Angielski / Miękka / 2014 / 275 str.

Dongsheng Duan

Opis

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.

Spis treści

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.

Blurb

Muscle disease represents an important health threat to the general population. For thousands of years, a cure has been deemed extremely remote, if not impossible, for many relentless muscle diseases such as Duchenne muscular dystrophy. The cloning of muscle disease genes and the prospect of introducing the normal gene to diseased muscle by gene therapy finally bring the hope of a cure. Tremendous progress has been made chasing the muscle gene therapy dream over the past two decades. Valuable information is scattered in the literature. This book represents the first compilation specifically dedicated to issues related to muscle gene therapy. It offers a much needed, up-to-date overview and perspective on the current strategies and status of muscle gene therapy. It covers the cutting edge development of a variety of fascinating strategies such as exon-skipping, AAV gene therapy, RNAi and stem cell approaches. With contributions from prominent investigators in the field, it provides a framework to translate bench science to clinical practice in the upcoming years. This book is a must-have for anyone who is interested in muscle gene therapy including established investigators, clinicians, post-doctoral fellows, graduate students, funding agencies, patients and their families and friends.