Molecular characterization of myotonic dystrophy fibroblast cell lines for use in small molecule screening

Summary: 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...

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Bibliographic Details
Main Authors: Jana R. Jenquin, Alana P. O’Brien, Kiril Poukalov, Yidan Lu, Jesus A. Frias, Hannah K. Shorrock, Jared I. Richardson, Hormoz Mazdiyasni, Hongfen Yang, Robert W. Huigens, III, David Boykin, Laura P.W. Ranum, John Douglas Cleary, Eric T. Wang, J. Andrew Berglund
Format: Article
Language:English
Published: Elsevier 2022-05-01
Series:iScience
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2589004222004680