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Therapeutic activity of modified U1 core spliceosomal particles
Modified U1 snRNAs bound to intronic sequences downstream of the 5′ splice site correct exon skipping caused by different types of mutations. Here we evaluate the therapeutic activity and structural requirements of these exon-specific U1 snRNA (ExSpeU1) particles. In a severe spinal muscular atrophy...
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| Publicado no: | Nat Commun |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4822034/ https://ncbi.nlm.nih.gov/pubmed/27041075 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms11168 |
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