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

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....

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Detalles Bibliográficos
Autores principales: Balestra, D, Faella, A, Margaritis, P, Cavallari, N, Pagani, F, Bernardi, F, Arruda, V R, Pinotti, M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BlackWell Publishing Ltd 2014
Materias:
Coagulation
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4238797/
https://www.ncbi.nlm.nih.gov/pubmed/24738135
http://dx.doi.org/10.1111/jth.12471

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