Targeting Toxic RNAs that Cause Myotonic Dystrophy Type 1 (DM1) with a Bisamidinium Inhibitor

[Image: see text] A working hypothesis for the pathogenesis of myotonic dystrophy type 1 (DM1) involves the aberrant sequestration of an alternative splicing regulator, MBNL1, by expanded CUG repeats, r(CUG)(exp). It has been suggested that a reversal of the myotonia and potentially other symptoms o...

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Hauptverfasser: Wong, Chun-Ho, Nguyen, Lien, Peh, Jessie, Luu, Long M., Sanchez, Jeannette S., Richardson, Stacie L., Tuccinardi, Tiziano, Tsoi, Ho, Chan, Wood Yee, Chan, H. Y. Edwin, Baranger, Anne M., Hergenrother, Paul J., Zimmerman, Steven C.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Chemical Society 2014
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4015652/
https://ncbi.nlm.nih.gov/pubmed/24702247
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/ja5012146
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