Muscular dystrophy in a dish: engineered human skeletal muscle mimetics for disease modeling and drug discovery.
Engineered in vitro models using human cells, particularly patient-derived induced pluripotent stem cells (iPSCs), offer a potential solution to issues associated with the use of animals for studying disease pathology and drug efficacy. Given the prevalence of muscle diseases in human populations, an engineered tissue model of human skeletal muscle could provide a biologically accurate platform to study basic muscle physiology, disease progression, and drug efficacy and/or toxicity. Such platforms could be used as phenotypic drug screens to identify compounds capable of alleviating or reversing congenital myopathies, such as Duchene muscular dystrophy (DMD). Here, we review current skeletal muscle modeling technologies with a specific focus on efforts to generate biomimetic systems for investigating the pathophysiology of dystrophic muscle.
Department of Bioengineering, University of Washington, Seattle, WA 98195, USA.
Alec S T
AST
Smith
Department of Bioengineering, University of Washington, Seattle, WA 98195, USA; Department of Pathology, University of Washington, Seattle, WA 98195, USA; Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA; Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA.
Jennifer
J
Davis
Institute for Cell Engineering, Department of Neurology, The Solomon H. Snyder Department of Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
Gabsang
G
Lee
Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA; Department of Rehabilitation Medicine, University of Washington, Seattle, WA 98195, USA.
David L
DL
Mack
Department of Bioengineering, University of Washington, Seattle, WA 98195, USA; Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA; Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA. Electronic address: deokho@uw.edu.
Deok-Ho
DH
Kim
D000818:Animals; D032701:Biomimetics; D055808:Drug Discovery; D006801:Humans; D008954:Models, Biological; D018482:Muscle, Skeletal / Q000502:physiology*; D009136:Muscular Dystrophies / Q000503:physiopathology*; D023822:Tissue Engineering