Significant advances are being made in the study of musculoskeletal disease. Where conditions like Duchenne muscular dystrophy (DMD) were once seen as untreatable, hope now swells as gene therapies advance into the market. Critical to the study and treatment of conditions like DMD is the use of physiologically relevant musculoskeletal disease models. As immortalised human muscle cell lines can be difficult to access, and animal myoblast cell lines are phenotypically distinct from their in vivo counterparts, many in the field are now turning to a more physiologically accurate source of cells: iPSC-derived myocytes [1].
bit.bio’s deterministic cell programming technology (known as opti-ox™) makes it possible to generate functional human skeletal muscle cells with unmatched lot-to-lot consistency from iPSCs on a nearly limitless scale (all without the challenges typically associated with myocyte differentiation). Both wild-type and disease-specific myocytes are readily available, enabling researchers to study skeletal myocytes in vitro with ever more translational power.
Leveraging genetically matched controls, human iPSC-derived skeletal myocytes containing DMD mutations can be used to screen prospective therapeutics, including exon-skipping therapies and other advanced drug modalities.
Strengthen your research with bit.bio’s human iPSC-derived skeletal myocytes.
Dr Rita Horvath, Director of Research in Genetics of Rare Neurological Disorders at the University of Cambridge discusses the mechanical and metabolic roles of myocytes in health and disease, experimental limitations and new opportunities offered by consistent, scalable human myocytes for muscle research and disease modelling.
Bernard, et al
bit.bio
2024
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Dr Will Bernard | Director of Cell Type Development | bit.bio
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Prof Hagan Bayley | University of Oxford
Dr Mark Kotter | Founder and CEO | bit.bio
1. Danisovic L, Culenova M, Csobonyeiova M (2018) Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy. Cells. 2018 Dec 7;7(12):253. doi: 10.3390/cells7120253. PMCID: PMC6315586 PMID: 30544588