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

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...

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Detalhes bibliográficos
Main Authors: Skordis, Leigh A., Dunckley, Matthew G., Yue, Baigong, Eperon, Ian C., Muntoni, Francesco
Formato: Artigo
Idioma:Inglês
Publicado em: The National Academy of Sciences 2003
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC153057/
https://ncbi.nlm.nih.gov/pubmed/12642665
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0633863100
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