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.

Autorentext
Martin K. (Casey) Childers, D.O., Ph.D. is a professor in the Department of Rehabilitation Medicine and Investigator at the Institute for Stem Cell & Regenerative Medicine, University of Washington. He is a graduate of Seattle Pacific University (B.A., Music Performance), Western University (D.O., Medicine, Osteopathic) and The University of Missouri (Ph.D., Physiology & Pharmacology; residency, Rehabilitation Medicine). The Childers' laboratory works in two areas of investigation. In a series of preclinical studies, they address the hurdles required for systemic gene replacement delivery for patients with X-Linked Myotubular Myopathy (XLMTM). In other studies, they use a "disease in a dish" approach with induced pluripotent stem (iPS) cells to study heart disease in patients with Duchenne muscular dystrophy (DMD). Dr. Childers clinical medicine practice at the University of Washington Medical Center is dedicated to serve patients with neuromuscular diseases.

Inhalt

Preface.- 1. Regenerative Medicine Approaches to Degenerative Muscle Diseases.- 2. An Overview of rAAV Vector Product Development for Gene Therapy.- 3. Gene Discovery in Congenital Myopathy.- 4. Stem Cell Transplantation for Degenerative Muscle.- 5. Spinal Cord Cellular Therapeutics Delivery: Device Design Considerations.- 6. Patient Derived Induced Pluripotent Stem Cells Provide a Regenerative Medicine Platform for Duchenne Muscular Dystrophy Heart Failure.- 7. Overview of Chemistry, Manufacturing and Controls (CMC) for Pluripotent Stem Cell-Based Therapies.- 8. Regenerative Rehabilitation: Synergizing Regenerative Medicine Therapies with Rehabilitation for Improved Muscle Regeneration in Muscle Pathologies.- 9. Practical Nutrition Guidelines for Individuals with Duchenne Muscular Dystrophy.- 10. Identifying Beneficial Drug Therapies for Muscle Disease Using Zebrafish.- 11. The Use of miRNAs in Muscle Diseases.- 12 Canine Inherited Dystrophinopathies and Centronuclear Myopathies.- Index.