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Design principles for bifunctional targeted oligonucleotide enhancers of splicing
Controlling the patterns of splicing of specific genes is an important goal in the development of new therapies. We have shown that the splicing of a refractory exon, SMN2 exon 7, could be increased in fibroblasts derived from patients with spinal muscular atrophy by using bifunctional targeted olig...
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| Autori principali: | , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Oxford University Press
2011
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3167598/ https://ncbi.nlm.nih.gov/pubmed/21602265 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkr152 |
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