In Vitro Evaluation of Exon Skipping in Disease-Specific iPSC-Derived Myocytes

Methods in Molecular Biology
Mingming ZhaoHidetoshi Sakurai

Abstract

Patient-derived disease-specific induced pluripotent stem cells (iPSCs) have opened the door to recreating pathological conditions in vitro using differentiation into diseased cells corresponding to each target tissue. To investigate muscular disease, we have established a myogenic differentiation protocol mediated by inducible MYOD1 expression that drives human iPSCs into myocytes. This highly reproducible differentiation protocol yields a homogenous skeletal muscle cell population, reaching efficiencies as high as 70-90%. Such high efficiency enables us to evaluate the efficacy of exon skipping in disease-specific myocytes. These disease-specific iPSC-derived myocytes can be applied not only for the validation of therapeutic efficacy of specific antisense oligonucleotide but also for the screening of exon skipping chemicals combined with the multiwell differentiation system.

Citations

Apr 2, 2019·Journal of Cellular and Molecular Medicine·María Del Carmen Ortuño-CostelaMaría Esther Gallardo
Dec 8, 2019·Nature Communications·Hiroyuki MorisakaTomoji Mashimo
Dec 31, 2020·Molecular Therapy. Methods & Clinical Development·Naoki YahataRyuji Hata
Feb 26, 2021·Pflügers Archiv : European journal of physiology·Kalina AndrysiakJózef Dulak

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