טוען...
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...
שמור ב:
| הוצא לאור ב: | Proc Natl Acad Sci U S A |
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| Main Authors: | , , , , , , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
National Academy of Sciences
2019
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| נושאים: | |
| גישה מקוונת: | 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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