Concepedia

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

Dario Balestra, Armida Faella, Paris Margaritis, Nicola Cavallari, Francesco Bernardi, Valder R. Arruda, Mirko Pinotti

Journal of Thrombosis and Haemostasis · 2013 · 47 citations · 27 references

DOIFull text

Open access

Abstract

Here we provide the first in vivo proof of-principle of the rescue of the expression of a splicing-defective F7 mutant by U1snRNAs, thus highlighting their therapeutic potential in coagulation disorders.

References

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