This book compiles andexplores cutting-edge research in degenerative skeletal disorders, such asDuchenne muscular dystrophy and congenital myopathy, and new stem-cell basedtherapies and gene replacement therapy. Twelve expertly-authored chapters navigatethe nuances of these treatments in an array of contexts and biological systems.The topics covered include: How are urine cells from a patient with Duchennemuscular dystrophy transformed into beating heart cells? What can reprogrammedcells tell us about heart muscle failure? What do gene mutations mean for thoseborn with a muscle disease? How are manufacturing methods applied to human stemcells? Does therapeutic exercise benefit those patients who receive engineeredlimb muscle? Is there practical advice about nutrition to enhance musclefunction for the Duchenne patient? Can microRNAs be useful to regeneratediseased muscle?
RegenerativeMedicine for Degenerative Muscle Diseases is ideal for scientistsand clinicians from varying disciplines in genetics, cell biology, virology,cell-based manufacturing, rehabilitation medicine, nutrition, veterinarymedicine and neurosurgery. The reader will see how transformative changes occurin medicine that can powerfully impact the future for patients suffering frominherited disorders affecting muscles of the body, including the heart.
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-Blc…