Abstract

In Vitro Modeling of Neuromuscular Diseases Using Human Induced Pluripotent Stem Cells

Masatoshi Suzuki, Associate Professor, Department of Comparative Biosciences and Stem Cell & Regenerative Medicine Center, University of Wisconsin-Madison

Neuromuscular diseases are caused by functional defects of skeletal muscles directly via muscle pathology or indirectly via the nervous system. Extensive studies have been performed to improve the outcomes of therapies; however, effective treatment strategies have not been fully established in any major neuromuscular disease. Human induced pluripotent stem cells (iPSCs) have a great capacity to differentiate into skeletal muscle progenitor cells (or know as myogenic progenitors) and skeletal myocytes for use in treating and modeling neuromuscular diseases. Further, recent innovations in bioengineering provide multifactorial and multidimensional controlled platforms for biomedical research beyond the traditional culture systems. Specifically, patient-derived iPSCs can be applied to differentiate into mature skeletal myotubes using newly featured cell culture systems such as two-dimensional (2D) skeletal-muscle-on-a-chip and three-dimensional (3D) skeletal muscle organoids. These bioengineering approaches can more closely mimic the native cellular environment and pathology in culture. Skeletal myotubes derived from patient-specific iPSC lines are a valuable resource for studying neuromuscular disease mechanisms and testing potential drug therapies.