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Molecular characterization of myotonic dystrophy fibroblast cell lines for use in small molecule screening
Myotonic dystrophy type 1 (DM1) and type 2 (DM2) are common forms of adult onset muscular dystrophy. Pathogenesis in both diseases is largely driven by production of toxic-expanded repeat RNAs that sequester MBNL RNA-binding proteins, causing mis-splicing. Given this shared pathogenesis, we hypothes...
Autores principales: | Jenquin, Jana R., O’Brien, Alana P., Poukalov, Kiril, Lu, Yidan, Frias, Jesus A., Shorrock, Hannah K., Richardson, Jared I., Mazdiyasni, Hormoz, Yang, Hongfen, Huigens, Robert W., Boykin, David, Ranum, Laura P.W., Cleary, John Douglas, Wang, Eric T., Berglund, J. Andrew |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9035709/ https://www.ncbi.nlm.nih.gov/pubmed/35479399 http://dx.doi.org/10.1016/j.isci.2022.104198 |
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