Regenerative Medicine for Degenerative Muscle Diseases (Stem Cell Biology and Regenerative Medicine)

This book compiles and explores cutting-edge research in degenerative skeletal disorders, such as Duchenne muscular dystrophy and congenital myopathy, and new stem-cell based therapies and gene replacement therapy. Twelve expertly-authored chapters navigate the nuances of these treatments in an array of contexts and biological systems. The topics covered include: How are urine cells from a patient with Duchenne muscular dystrophy transformed into beating heart cells? What can reprogrammed cells tell us about heart muscle failure? What do gene mutations mean for those born with a muscle disease? How are manufacturing methods applied to human stem cells? Does therapeutic exercise benefit those patients who receive engineered limb muscle? Is there practical advice about nutrition to enhance muscle function for the Duchenne patient? Can microRNAs be useful to regenerate diseased muscle? __Regenerative Medicine for Degenerative Muscle Diseases__ is ideal for scientists and clinicians from varying disciplines in genetics, cell biology, virology, cell-based manufacturing, rehabilitation medicine, nutrition, veterinary medicine and neurosurgery. The reader will see how transformative changes occur in medicine that can powerfully impact the future for patients suffering from inherited disorders affecting muscles of the body, including the heart. "This book compiles and explores cutting-edge research in degenerative skeletal disorders, such as Duchenne muscular dystrophy and congenital myopathy, and new stem-cell based therapies and gene replacement therapy. Twelve expertly-authored chapters navigate the nuances of these treatments in an array of contexts and biological systems. The topics covered include: How are urine cells from a patient with Duchenne muscular dystrophy transformed into beating heart cells? What can reprogrammed cells tell us about heart muscle failure? What do gene mutations mean for those born with a muscle disease? How are manufacturing methods applied to human stem cells? Does therapeutic exercise benefit those patients who receive engineered limb muscle? Is there practical advice about nutrition to enhance muscle function for the Duchenne patient? Can microRNAs be useful to regenerate diseased muscle? Regenerative Medicine for Degenerative Muscle Diseases is ideal for scientists and clinicians from varying disciplines in genetics, cell biology, virology, cell-based manufacturing, rehabilitation medicine, nutrition, veterinary medicine and neurosurgery. The reader will see how transformative changes occur in medicine that can powerfully impact the future for patients suffering from inherited disorders affecting muscles of the body, including the heart"--Provided by publisher Front Matter....Pages i-xiii Regenerative Medicine Approaches to Degenerative Muscle Diseases....Pages 1-20 An Overview of rAAV Vector Product Development for Gene Therapy....Pages 21-37 Gene Discovery in Congenital Myopathy....Pages 39-83 Stem Cell Transplantation for Degenerative Muscle Diseases....Pages 85-108 Spinal Cord Cellular Therapeutics Delivery: Device Design Considerations....Pages 109-127 Patient-Derived Induced Pluripotent Stem Cells Provide a Regenerative Medicine Platform for Duchenne Muscular Dystrophy Heart Failure....Pages 129-155 Overview of Chemistry, Manufacturing, and Controls (CMC) for Pluripotent Stem Cell-Based Therapies....Pages 157-203 Regenerative Rehabilitation: Synergizing Regenerative Medicine Therapies with Rehabilitation for Improved Muscle Regeneration in Muscle Pathologies....Pages 205-224 Practical Nutrition Guidelines for Individuals with Duchenne Muscular Dystrophy....Pages 225-279 Identifying Therapies for Muscle Disease Using Zebrafish....Pages 281-293 miRNAs in Muscle Diseases....Pages 295-307 Canine-Inherited Dystrophinopathies and Centronuclear Myopathies....Pages 309-329 Back Matter....Pages 331-336