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Article

Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates <i>SMN2</i> gene expression in patient fibroblasts

17 Mar 2003

Abstract excerpt

The multiplicity of proteins compared with genes in mammals owes much to alternative splicing. Splicing signals are so subtle and complex that small perturbations may allow the production of new mRNA variants. However, the flexibility of splicing can also be a liability, and several genetic diseases result from single-base changes that cause exons to be skipped during splicing. Conventional oligonucleotide...

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PMID: 12642665
DOI: 10.1073/pnas.0633863100

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