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SELECTBIO Conferences High-Content and Phenotypic Screening Europe 2018


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.

Add to Calendar ▼2018-05-24 00:00:002018-05-25 00:00:00Europe/LondonHigh-Content and Phenotypic Screening Europe 2018High-Content and Phenotypic Screening Europe 2018 in Cambridge, UKCambridge,