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Pentamidine reverses the splicing defects associated with myotonic dystrophy
Myotonic dystrophy (DM) is a genetic disorder caused by the expression (as RNA) of expanded CTG or CCTG repeats. The alternative splicing factor MBNL1 is sequestered to the expanded RNA repeats, resulting in missplicing of a subset of pre-mRNAs linked to symptoms found in DM patients. Current data s...
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| Main Authors: | , , , , |
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| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
National Academy of Sciences
2009
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| Teme: | |
| Online dostop: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2774031/ https://ncbi.nlm.nih.gov/pubmed/19822739 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0903234106 |
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