Interaction of hnRNPA1/A2 and DAZAP1 with an Alu-derived intronic splicing enhancer regulates ATM aberrant splicing.

by Tibor Pastor, Franco Pagani
Published 2011-01-01

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

New lysosomal acid lipase gene mutants explain the phenotype ofWolman disease and cholesteryl ester storage disease

by Franco Pagani, Rajalakshmi Pariyarath, Rodolfo Garcia, Cristiana Stuani, Alberto B. Burlina, Giacomo Ruotolo, Marco Rabusin, Francisco E. Baralle
Published 1998-07-01

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

by Dario Balestra, Daniela Scalet, Mattia Ferrarese, Silvia Lombardi, Nicole Ziliotto, Chrystal C. Croes, Naomi Petersen, Piter Bosma, Federico Riccardi, Franco Pagani, Mirko Pinotti, Stan F. J. van de Graaf
Published 2020-03-01