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An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects
A significant proportion of disease-causing mutations affect precursor-mRNA splicing, inducing skipping of the exon from the mature transcript. Using F9 exon 5, CFTR exon 12 and SMN2 exon 7 models, we characterized natural mutations associated to exon skipping in Haemophilia B, cystic fibrosis and s...
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| Main Authors: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2012
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3349419/ https://ncbi.nlm.nih.gov/pubmed/22362925 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/dds045 |
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