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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 >>
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International Society on Thrombosis and Haemostasis (ISTH),
ISTH2020
pp: 00-00, Anno: 2020 |
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Rescue of a FVIII splicing variant with engineered U1snRNAs
Peretto, Laura; Tarantino, Rebecca; Borchiellini, Alessandra; Bernardi, Francesco; Follenzi, Antonia; Pinotti, Mirko; Balestra, Dario
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Wiley,
Research and Practice in Thrombosis and Haemostasis
pp: 95-95, Anno: 2022 |
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Base and Prime editing of DNA as a new therapeutic option for Hemophilia A
Tonetto, Elena; Pignani, Silvia; Roman, Giacomo; Follenzi, Antonia; Bernardi, Francesco; Liu, David; Pinotti, Mirko; Balestra, Dario
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Wiley,
Research and Practice in Thrombosis and Haemostasis
pp: 95-95, Anno: 2022 |
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Rescue of a hemophilia A-causing FVIII splicing variant via engineered U1SNRNAS
Peretto, Laura; Tarantino, Rebecca; Borchiellini, Alessandra; Bernardi, Francesco; Follenzi, Antonia; Pinotti, Mirko; Balestra, Dario
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Pagepress,
XXVII Congresso Nazionale SISET Perugia 2-5 novembre 2022
Vol. 1, No. Supplement 1, pp: 47-47, Anno: 2022 |
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New Genome Editing Approaches: Base And Prime Editing To Revert Hemophilia A-Causing Point Mutations
Tonetto, E.; Pignani, S.; Roman, G.; Follenzi, A.; Bernardi, F.; Liu, D.; Pinotti, M.; Balestra, D.
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Pagepress,
BLEEDING, THROMBOSIS AND VASCULAR BIOLOGY
pp: 46-47, Anno: 2022 |
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Deep molecular mechanisms of F8 exon 19 variants and translational approaches in Hemophilia A
Lombardi, Silvia; Peretto, Laura; Merlin, Simone; Follenzi, Antonia; Mcvey, John H.; Maestri, Iva; Bernardi, Francesco; Pinotti, Mirko; Balestra, Dario
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Ferrara Storti foundation,
Abstract Book of International Conference Advances in Haemostasis and Bleeding Disorders
Vol. 106, No. Suppl 1, pp: 25-25, Anno: 2021 |
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Contribution of asialoglycoprotein receptor ASGR2 5’ UTR polymorphisms to full-length FVIII concentrate pharmacokinetics
Lunghi, B.; Morfini, M.; Martinelli, N.; Frusconi, S.; Balestra, D.; Branchini, A.; Linari, S.; Marchetti, G.; Castaman, G.; Bernardi, F.
dettagli >>
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SIMTI,
XXVI Congresso Nazionale della Società Italiana per lo Studio dell'Emostasi e della Trombosi - SISET
Vol. 18, No. Suppl 4, pp: S431-S432, Anno: 2020 |
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The asialoglycoprotein receptor ASGR2 5’ UTR polymorphisms influence several parameters of full-length FVIII concentrate pharmacokinetics
Lunghi, B.; Morfini, M.; Martinelli, N.; Frusconi, S.; Balestra, D.; Branchini, A.; Linari, S.; Marchetti, G.; Castaman, G.; Bernardi, F.
dettagli >>
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SIMTI,
XVII Convegno Triennale sui Problemi Clinici e Sociali dell’Emofilia e delle Malattie Emorragiche Congenite
Vol. 18, No. Suppl 3, pp: S350-S351, Anno: 2020 |
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Rescue of multiple Haemophilia A-causing mutations by a single ExSpeU1: the importance of the genomic context
Balestra, Dario; Lombardi, Silvia; Leo, Gabriele; Calanchi, Irene; Bernardi, Francesco; Pinotti, Mirko
dettagli >>
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Wiley,
Special Issue: Abstracts of the XXVII Congress of the International Society on Thrombosis and Haemostasis
Vol. 3, No. S1, pp: 313-313, Anno: 2019 |
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Exon-Specific U1snRNA-Mediated Rescue of Splicing and Missense Changes in Hemophilia A
Lombardi, Silvia; Leo, Gabriele; Maestri, Iva; Bernardi, Francesco; Pinotti, Mirko; Mcvey, John Henderson; Balestra, Dario
dettagli >>
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Wiley,
Special Issue: Abstracts of the XXVII Congress of the International Society on Thrombosis and Haemostasis
Vol. 3, No. S1, pp: 105-105, Anno: 2019 |
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Secretion of wild-type factor IX upon readthrough over F9 pre-peptide nonsense mutations causing hemophilia B
Ferrarese, Mattia; Testa, Maria Francesca; Balestra, Dario; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
dettagli >>
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Walter Ageno, Anna Falanga, Gianccarlo Castaman,
XXV Congresso Nazionale della Società Italiana per lo Studio dell'Emostasi e della Trombosi (SISET) - Oral Commincations
Vol. 16, No. Supplement 4, pp: 456-456, Anno: 2018 |
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The chaperone-like compound sodium phenylbutyrate improves intracellular trafficking, secretion and coagulant activity of factor IX impaired by the frequent p.R294Q mutation
Pignani, Silvia; Todaro, Alice; Ferrarese, Mattia; Marchi, Saverio; Lombardi, Silvia; Balestra, Dario; Pinton, Paolo; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
dettagli >>
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Walter Ageno, Anna Falanga, Gianccarlo Castaman,
XXV Congresso Nazionale della Società Italiana per lo Studio dell'Emostasi e della Trombosi (SISET) - Oral Commincations
Vol. 16, No. Supplement 4, pp: 418-418, Anno: 2018 |
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A strategy with chaperone-like compounds to restore expression of factor IX variants affected by frequent missense mutations causing hemophilia B
Pignani, Silvia; Todaro, Alice; Ferrarese, Mattia; Marchi, Saverio; Lombardi, Silvia; Balestra, Dario; Pinton, Paolo; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
dettagli >>
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Wolters Kluwer,
Abstract book of the 23rd Congress of the European Hematology Association
Vol. 2, No. S1, pp: 393-393, Anno: 2018 |
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The carboxyl-terminal region of human coagulation factor X as a novel naturally-occuring linker for fusion strategies
Ferrarese, Mattia; Pignani, Silvia; Lombardi, Silvia; Balestra, Dario; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
dettagli >>
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Walter Ageno, Anna Falanga, Gianccarlo Castaman,
.XXV Congresso Nazionale della Società Italiana per lo Studio dell'Emostasi e della Trombosi (SISET). Oral Commincations
Vol. 16, No. Supplement 4, pp: 418-418, Anno: 2018 |
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Rescue of missense and splicing mutations in Haemophilia A by a unique Exon Specific U1snRNA
Balestra, Dario; Scalet, Daniela; Leo, Gabriele; Donadon, I.; Lombardi, Silvia; Bernardi, Francesco; Pinotti, M.
dettagli >>
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Edizioni SIMTI,
XXV Congresso Nazionale della Società Italiana per lo Studio dell'Emostasi e della Trombosi (SISET). Oral Commincations
Vol. 16, No. S4, pp: 454-454, Anno: 2018 |
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MOLECULAR MECHANISMS AND THERAPEUTIC APROACHES FOR RESTORATION OF mRNA TRANSCRIPTION, MATURATION AND TRANSLATION IN INHERITED COAGULATION FACTOR DEFICIENCIES
Balestra, Dario; Bovolenta, Matteo; Branchini, Alessio; Pinotti, Mirko; Bernardi, Francesco
dettagli >>
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Anna Falanga,
BLOOD TRANSFUSION
Vol. 14, No. S5, pp: 694-706, Anno: 2016 |
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Correction of aberrant splicing causing haemophilia B through the combination of compensatory U1snRNAs and antisense oligonucleotides
Scalet, Daniela; Balestra, Dario; Barbon, Elena; Cavallari, Nicola; Perrone, Daniela; Bernardi, Francesco; Pinotti, Mirko
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WILEY-BLACKWELL,
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
pp: 210-210, Anno: 2015 |
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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
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WILEY-BLACKWELL,
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
pp: 208-209, Anno: 2015 |
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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 >>
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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 |
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Delivery of a modified U1 small nuclear RNA alleviates splicing-defective coagulation Factor VII expression in mouse models
Balestra, Dario; A., Faella; N., Cavallari; P., Margaritis; F., Pagani; Bernardi, Francesco; V. R., Arruda; Pinotti, Mirko
dettagli >>
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JOURNAL OF THROMBOSIS AND HAEMOSTASIS
Vol. 2, No. 11, pp: 194-195, Anno: 2013 |
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