Regenerative Medicine for Degenerative Muscle Diseases

1. Front Matter

   Pages i-xiii

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2. Regenerative Medicine Approaches to Degenerative Muscle Diseases

   • Martin K. Childers, Zejing Wang

   Pages 1-20

3. An Overview of rAAV Vector Product Development for Gene Therapy

   • Richard O. Snyder

   Pages 21-37

4. Gene Discovery in Congenital Myopathy

   • Laura L. Smith, Vandana A. Gupta, Alan H. Beggs

   Pages 39-83

5. Stem Cell Transplantation for Degenerative Muscle Diseases

   • Berkcan Akpinar, Elizabeth C. Stahl, Aiping Lu, Johnny Huard

   Pages 85-108

6. Spinal Cord Cellular Therapeutics Delivery: Device Design Considerations

   • Khalid Medani, Jonathan Riley, Jason Lamanna, Nicholas Boulis

   Pages 109-127

7. Patient-Derived Induced Pluripotent Stem Cells Provide a Regenerative Medicine Platform for Duchenne Muscular Dystrophy Heart Failure

   • Xuan Guan, David Mack, Martin K. Childers

   Pages 129-155

8. Overview of Chemistry, Manufacturing, and Controls (CMC) for Pluripotent Stem Cell-Based Therapies

   • Amy Lynnette Van Deusen, Michael Earl McGary

   Pages 157-203

9. Regenerative Rehabilitation: Synergizing Regenerative Medicine Therapies with Rehabilitation for Improved Muscle Regeneration in Muscle Pathologies

   • Kristen Stearns-Reider, Fabrisia Ambrosio

   Pages 205-224

10. Practical Nutrition Guidelines for Individuals with Duchenne Muscular Dystrophy

    • Zoe E. Davidson, Greg Rodden, Davi A. G. Mázala, Cynthia Moore, Carol Papillon, Angela J. Hasemann et al.

    Pages 225-279

11. Identifying Therapies for Muscle Disease Using Zebrafish

    • Elizabeth U. Parker, Lisa Maves

    Pages 281-293

12. miRNAs in Muscle Diseases

    • Diem-Hang Nguyen-Tran, Hannele Ruohola-Baker

    Pages 295-307

13. Canine-Inherited Dystrophinopathies and Centronuclear Myopathies

    • Joe N. Kornegay, Martin K. Childers

    Pages 309-329

14. Back Matter

    Pages 331-336

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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.

• Cellular therapeutics
• Muscle disease and disorder
• Regenerative medicine
• Skeletal muscle
• Stem cell-based therapies

• Department of Rehabilitation Medicine, University of Washington Department of Rehabilitation Medicine, WINSTON SALEM, USA

  Martin K. Childers

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.

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