TY - JOUR
T1 - Muscular dystrophy in a dish
T2 - engineered human skeletal muscle mimetics for disease modeling and drug discovery
AU - Smith, Alec S.T.
AU - Davis, Jennifer
AU - Lee, Gabsang
AU - Mack, David L.
AU - Kim, Deok Ho
N1 - Funding Information:
This work was supported by Muscular Dystrophy Association (MDA) research grants to D-H.K. (Award ID 255907) and to G.L. (Award ID 381465), as well as a National Institutes of Health R21 Grant ( R21AR064395 ) (to D-H.K.), a New York Stem Cell Foundation award (to G.L.), and an International Collaborative R&D Program Grant with KIAT funded by the MOTIE (N0000894) awarded to D-H.K. D-H.K also thanks the Department of Bioengineering at the University of Washington for the new faculty startup fund.
Publisher Copyright:
© 2016 Elsevier Ltd
PY - 2016/9/1
Y1 - 2016/9/1
N2 - 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.
AB - 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.
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U2 - 10.1016/j.drudis.2016.04.013
DO - 10.1016/j.drudis.2016.04.013
M3 - Review article
C2 - 27109386
AN - SCOPUS:84964989160
VL - 21
SP - 1387
EP - 1398
JO - Drug Discovery Today
JF - Drug Discovery Today
SN - 1359-6446
IS - 9
ER -