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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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Detalles Bibliográficos
Autores principales:	Owen, Nicholas, Zhou, Haiyan, Malygin, Alexey A., Sangha, Jason, Smith, Lindsay D., Muntoni, Francesco, Eperon, Ian C.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2011
Materias:	RNA
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3167598/ https://www.ncbi.nlm.nih.gov/pubmed/21602265 http://dx.doi.org/10.1093/nar/gkr152

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