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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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| Pubblicato in: | Nat Commun |
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| Autori principali: | , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Nature Publishing Group
2016
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| Soggetti: | |
| Accesso online: | 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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