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

- Animal models can be poor mimics of healthy and diseased skeletal muscle phenotypes.
- Engineered muscle tissues offer alternative systems to study dystrophic pathology.
- Engineered muscle maturation is required to ensure models mimic in vivo physiology.
- This review covers strategies for promoting engineered muscle development.
- The effect such models could have muscular dystrophy therapies is also discussed.

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.