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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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
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