Drug Discov Today. 2016 Apr 22. pii: S1359-6446(16)30118-0. doi: 10.1016/j.drudis.2016.04.013. [Epub ahead of print]

Muscular dystrophy in a dish: engineered human skeletal muscle mimetics for disease modeling and drug discovery.

Smith AS¹, Davis J², Lee G³, Mack DL⁴, Kim DH⁵.

Abstract

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.