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Article

An engineered U1 small nuclear RNA rescues splicing‐defective coagulation F7 gene expression in mice

30 Nov 2013

Abstract excerpt

BACKGROUND: The ability of the spliceosomal small nuclear RNA U1 (U1snRNA) to rescue pre-mRNA splicing impaired by mutations makes it an attractive therapeutic molecule. Coagulation factor deficiencies due to splicing mutations are relatively frequent and could therefore benefit from this strategy. However, the effects of U1snRNAs in vivo remain unknown. OBJECTIVES: To assess the rescue of the F7 c.859+5G>A...

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PMID: 24738135
DOI: 10.1111/jth.12471

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