Applications of Human Pluripotent Stem Cells for Neuromuscular Diseases: Advances and Challenges
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 pluripotent stem
cells, such as embryonic stem cells and induced pluripotent stem cells,
have a great capacity to differentiate into skeletal muscle
progenitor/stem cells (or called myogenic progenitors) and skeletal
myocytes for use in treating and modeling neuromuscular diseases.
Particularly, recent advances allow us to create patient-derived stem
cells, which can be used as a unique platform for comprehensive study of
disease mechanisms and drug screening in vitro. In the last decade, a
number of protocols (including ours) have been established for
derivation of skeletal muscle cells from human pluripotent stem cells.
These protocols have been developed by following the process of
myogenesis controlled by the expression of transcription factors and
signaling molecules to specify cellular lineage during muscle
development. This presentation overviews recent progress of the
protocols for skeletal muscle derivation using human pluripotent stem
cells. We also discuss the potential limitations and promise of these
approaches for future applications of myogenic progenitors and myocytes
in neuromuscular diseases.
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