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

Alec S T Smith, Jennifer Davis, Gabsang Lee, David L. Mack, Deok Ho Kim

Research output: Contribution to journalReview article

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.

Original languageEnglish (US)
Pages (from-to)1387-1398
Number of pages12
JournalDrug Discovery Today
Volume21
Issue number9
DOIs
StatePublished - Sep 1 2016

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

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