Regenerative Medicine Approaches to Degenerative Muscle Diseases

  • Martin K. ChildersEmail author
  • Zejing Wang
Part of the Stem Cell Biology and Regenerative Medicine book series (STEMCELL)


The emerging multidisciplinary field of regenerative medicine aims to develop new technology to repair and replace cells, tissues, and organs. Regenerative medicine holds the promise of regenerating damaged tissues and organs in the body by either replacing damaged tissue or by stimulating the body’s own repair mechanisms to heal previously irreparable tissues or organs. Regenerative approaches address the root cause of disease and offer prospects of tissue repair previously unthinkable. For degenerative muscular disorders, most fall into the category of rare inherited diseases. To cure inherited muscular disorders, recent attention has focused on the transfer of normal genes to correct mutant diseased genes. This chapter discusses cell- and vector-mediated gene therapy technology under development for various inherited disorders of skeletal muscle.


Regenerative medicine Stem cells Gene therapy Myopathy Muscular dystrophy Adeno-associated virus 


Conflict of Interest Statement

MC is an inventor on patents related to recombinant AAV technology. MC owns stock options in a biotechnology company commercializing AAV for gene therapy applications. To the extent that the work in this manuscript increases the value of these commercial holdings, MC has a conflict of interest.


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Copyright information

© Springer Science+Business Media New York 2016

Authors and Affiliations

  1. 1.Department of Rehabilitation MedicineUniversity of WashingtonSeattleUSA
  2. 2.Institute for Stem Cell and Regenerative Medicine, School of MedicineUniversity of WashingtonSeattleUSA
  3. 3.Medicine/Clinical ResearchUniversity of Washington/Fred Hutchinson Cancer Research CenterSeattleUSA

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