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Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines
ISCU myopathy is an inherited disease that primarily affects individuals of northern Swedish descent who share a single point mutation in the fourth intron of the ISCU gene. The current study shows correction of specific phenotypes associated with disease following treatment with an antisense oligon...
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| Veröffentlicht in: | Hum Mol Genet |
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| Hauptverfasser: | , , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Oxford University Press
2016
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6078641/ https://ncbi.nlm.nih.gov/pubmed/28007899 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddw338 |
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