Abstract
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.
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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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Childers, M.K., Wang, Z. (2016). Regenerative Medicine Approaches to Degenerative Muscle Diseases. In: Childers, M. (eds) Regenerative Medicine for Degenerative Muscle Diseases. Stem Cell Biology and Regenerative Medicine. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3228-3_1
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