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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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| Publicado no: | Hum Mol Genet |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2016
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| Assuntos: | |
| Acesso em linha: | 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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