Generation of human iPSC-derived Duchenne muscular dystrophy skeletal myocytes suitable for 3D functional studies and investigating methods for dystrophin restoration

Skeletal myocytes play several roles in biological processes ranging from limb movement to the regulation of nutritional homeostasis and are implicated in the pathophysiology of diseases such as muscular dystrophies and metabolic disorders. There is a pressing need for reliable, easily accessible models of human skeletal muscle to improve investigations into these diseases, and to facilitate the generation of new therapeutics. 

This poster explains the development of human iPSC-derived ioSkeletal Myocytes and Duchenne muscular dystrophy (DMD) models with disease-relevant exon deletions that show reduced expression of dystrophin, and demonstrates functionality in 2D culture and 3D muscle microtissues.

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• How a DMD in vitro cell model was generated in human iPSC-derived skeletal myocytes 
• Data demonstrating dystrophin mRNA and protein expression restoration using ASO-mediated exon skipping in the DMD Exon 44 Deletion disease model
• Data showing reduced contractile function of the disease models compared to the wild type, genetically matched control in 2D culture and in 3D muscle microtissues cultured on Bi/ond’s MUSbit™ microchip