Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model

Myotonic dystrophy type 1 (DM1) is an incurable neuromuscular disorder caused by an expanded CTG repeat that is transcribed into r(CUG)(exp). The RNA repeat expansion sequesters regulatory proteins such as Muscleblind-like protein 1 (MBNL1), which causes pre-mRNA splicing defects. The disease-causin...

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Foilsithe in:Proc Natl Acad Sci U S A
Main Authors: Angelbello, Alicia J., Rzuczek, Suzanne G., Mckee, Kendra K., Chen, Jonathan L., Olafson, Hailey, Cameron, Michael D., Moss, Walter N., Wang, Eric T., Disney, Matthew D.
Formáid: Artigo
Teanga:Inglês
Foilsithe: National Academy of Sciences 2019
Ábhair:
Biological Sciences
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6475439/
https://ncbi.nlm.nih.gov/pubmed/30926669
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1901484116
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