An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants

por Balestra, Dario, Scalet, Daniela, Pagani, Franco, Rogalska, Malgorzata Ewa, Mari, Rosella, Bernardi, Francesco, Pinotti, Mirko
Publicado 2016

Regulation of a strong F9 cryptic 5′ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides

por Balestra, Dario, Barbon, Elena, Scalet, Daniela, Cavallari, Nicola, Perrone, Daniela, Zanibellato, Silvia, Bernardi, Francesco, Pinotti, Mirko
Publicado 2015

The Somatic FAH c.1061C>A Change Counteracts the Frequent FAH c.1062+5G>A Mutation and Permits U1snRNA-Based Splicing Correction

por Scalet, Daniela, Sacchetto, Claudia, Bernardi, Francesco, Pinotti, Mirko, van de Graaf, Stan F.J., Balestra, Dario
Publicado 2018

A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I

por Balestra, Dario, Scalet, Daniela, Ferrarese, Mattia, Lombardi, Silvia, Ziliotto, Nicole, C. Croes, Chrystal, Petersen, Naomi, Bosma, Piter, Riccardi, Federico, Pagani, Franco, Pinotti, Mirko, van de Graaf, Stan F. J.
Publicado 2020