The F7 p.Val22Ile missense mutation affects splicing and can be counteracted by a compensatory U1snRNA
Ferraresi, P.; Rizzotto, L.; Branchini, A.; Baroni, M.; Balestra, D.; Bernardi, F.; Pagani, F.; Pinotti, M.     dettagli >> 		International Society on Thrombosis and Haemostasis (ISTH), ISTH2020
pp: 00-00, Anno: 2020
RNA−based therapeutic approaches for blood coagulation factor deficiencies caused by splicing mutations
Pinotti, Mirko; Pagani, Franco; Balestra, Dario; Baroni, Marcello; Branchini, Alessio; Bussani, E.; Canella, Alessandro; Campioni, Matteo; Cavallari, N.; Dal Mas, A.; Fernandez, E.; Ferraresi, Paolo; Mattioli, C.     		Comitato Telethon Fondazione Onlus, XVI Scientific Convention
pp: 98-98, Anno: 2011
AN EXON-SPECIFIC U1 SMALL NUCLEAR RNA (snRNA) STRATEGY TO CORRECT SPLICING MUTATIONS ASSOCIATED TO HEMOPHILIA B
Balestra, Dario; Cavallari, Nicola; E., Fernandez Alanis; A., Dal Mas; M. E., Rogalska; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko     dettagli >> 		Claudio Velati, BLOOD TRANSFUSION
Vol. 10, Supplement no. 4, No. 1, pp: 46-46, Anno: 2012
A unique exon specific U1snRNA rescues different haemophilia B - causing splicing-defective factor IX variants in mice
Balestra, Dario; Scalet, Daniela; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko     		WILEY-BLACKWELL, JOURNAL OF THROMBOSIS AND HAEMOSTASIS
pp: 208-209, Anno: 2015
RNA-based therapeutic approaches for blood coagulation factor deficiencies caused by a splicing mutations
Balestra, Dario; Fernandez, Eugenio; Dal Mas, Andrea; Cavallari, Nicola; Barbon, Elena; Bussani, Erica; Ferraresi, Paolo; Canella, Alessandro; Branchini, Alessio; Campioni, Matteo; Baroni, Marcello; Mattioli, Chiara; Pagani, Franco; Pinotti, Mirko     dettagli >> 		Pinelli Printing S.r.l., Via E. Fermi, 8 - 20096 Seggiano di Pioltello (MI), XVII Convention Scientifica 11-13 Marzo 2013
pp: 112-112, Anno: 2013
“Compensatory” aberrant splicing supports residual expression levels in severe coagulation factor VII deficiency
Cavallari, N.; Balestra, Dario; Rizzotto, Lara; Branchini, Alessio; Maestri, Iva; Chuansumrit, A.; Sasanakul, W.; Mariani, Guglielmo; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko     dettagli >> 		International Society on Thrombosis and Haemostasis, JOURNAL OF THROMBOSIS AND HAEMOSTASIS
pp: 18-19, Anno: 2011
The complete impairment of factor VII gene expression by the IVS6+1g/t mutation is compatible with a severe but not lethal bleeding disorder
Cavallari, Nicola; Balestra, Dario; Rizzotto, Lara; A., Chuamsunrit; Mariani, Guglielmo; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko     dettagli >> 		Claudio Velati, BLOOD TRANSFUSION
Vol. 8, No. 4, pp: 43-43, Anno: 2010
Rescue of coagulation factor VII mRNA processing and protein function by engineered U1+5A snRNA
Balestra, Dario; Cavallari, Nicola; Maestri, Iva; Mari, Rosella; Rizzotto, Lara; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko     dettagli >> 		Claudio Velati, BLOOD TRANSFUSION
Vol. 8, No. 4, pp: 23-24, Anno: 2010
U1-snRNA-mediated rescue of mRNA processing in severe factor VII deficiency
Pinotti, Mirko; Balestra, Dario; Rizzotto, Lara; Cavallari, Nicola; Pagani, Franco; Bernardi, Francesco     dettagli >> 		Jan Cools, HAEMATOLOGICA
Vol. 93, No. 3, pp: 33-33, Anno: 2008