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Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts.

By L.A. Skordis, M.G. Dunckley, B.-G. Yue, I.C. Eperon and F. Muntoni
Year: 2003
DOI identifier: 10.1073/pnas.0633863100
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