Therapeutic activity of modified U1 core spliceosomal particles

by Malgorzata Ewa Rogalska, Mojca Tajnik, Danilo Licastro, Erica Bussani, Luca Camparini, Chiara Mattioli, Franco Pagani
Published 2016-04-01

Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein Function.

by Mojca Tajnik, Malgorzata Ewa Rogalska, Erica Bussani, Elena Barbon, Dario Balestra, Mirko Pinotti, Franco Pagani
Published 2016-05-01

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

by Dario Balestra, Daniela Scalet, Franco Pagani, Malgorzata Ewa Rogalska, Rosella Mari, Francesco Bernardi, Mirko Pinotti
Published 2016-01-01