Sfoglia per Autore
A unique exon specific U1snRNA rescues different haemophilia B - causing splicing-defective factor IX variants in mice
file con accesso da definire2015 Balestra, Dario; Scalet, Daniela; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko
Genetic determinants of activated factor VII antithrombin complex plasma concentration include tissue factor, factor VII and endothelial protein C receptor gene variants
file con accesso da definire2015 Sartori, F; Lunghi, Barbara; Tosi, F; Guarini, P; Scalet, Daniela; Baroni, Marcello; Marchetti, Giovanna; Woodhams, B; Girelli, D; Olivieri, O; Bernardi, Francesco; Martinelli, N.
Correction of aberrant splicing causing haemophilia B through the combination of compensatory U1snRNAs and antisense oligonucleotides
file con accesso da definire2015 Scalet, Daniela; Balestra, Dario; Barbon, Elena; Cavallari, Nicola; Perrone, Daniela; Bernardi, Francesco; Pinotti, Mirko
Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides
2015 Balestra, Dario; Barbon, Elena; Scalet, Daniela; Cavallari, Nicola; Perrone, Daniela; Zanibellato, Silvia; Bernardi, Francesco; Pinotti, Mirko
An exon-specific U1snRNA induces a robust factor IX activity in mice expressing multiple human FIX splicing mutants
2016 Balestra, Dario; Scalet, Daniela; Pagani, Franco; Rogalska, Malgorzata Ewa; Mari, Rosella; Bernardi, Francesco; Pinotti, Mirko
Regulation of exon definition by intrinsic elements and by combination of tailored U1snRNA with antisense oligonucleotides
2016 Scalet, Daniela
Exploring Splicing-Switching Molecules For Seckel Syndrome Therapy
2017 Scalet, Daniela; Balestra, Dario; Rohban, Sara; Bovolenta, Matteo; Perrone, Daniela; Bernardi, Francesco; Campaner, Stefano; Pinotti, Mirko
Rescue of missense and splicing mutations in Haemophilia A by a unique Exon Specific U1snRNA
2018 Balestra, Dario; Scalet, Daniela; Leo, Gabriele; Donadon, I.; Lombardi, Silvia; Bernardi, Francesco; Pinotti, M.
The somatic FAH C.1061C>A change counteracts the frequent FAH c.1062+5G>A mutation and permits U1snRNA-based splicing correction
2018 Scalet, Daniela; Sacchetto, Claudia; Bernardi, Francesco; Pinotti, Mirko; Van De Graaf, Stan F. J.; Balestra, Dario
Disease-causing variants of the conserved+2T of 5 ' splice sites can be rescued by engineered U1snRNAs
2019 Scalet, Daniela; Maestri, Iva; Branchini, Alessio; Bernardi, Francesco; Pinotti, Mirko; Balestra, Dario
A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I
2020 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.
| Titolo | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|
| A unique exon specific U1snRNA rescues different haemophilia B - causing splicing-defective factor IX variants in mice | 2015 | Balestra, Dario; Scalet, Daniela; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko | file con accesso da definire |
| Genetic determinants of activated factor VII antithrombin complex plasma concentration include tissue factor, factor VII and endothelial protein C receptor gene variants | 2015 | Sartori, F; Lunghi, Barbara; Tosi, F; Guarini, P; Scalet, Daniela; Baroni, Marcello; Marchetti, G...iovanna; Woodhams, B; Girelli, D; Olivieri, O; Bernardi, Francesco; Martinelli, N. | file con accesso da definire |
| Correction of aberrant splicing causing haemophilia B through the combination of compensatory U1snRNAs and antisense oligonucleotides | 2015 | Scalet, Daniela; Balestra, Dario; Barbon, Elena; Cavallari, Nicola; Perrone, Daniela; Bernardi, F...rancesco; Pinotti, Mirko | file con accesso da definire |
| Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides | 2015 | Balestra, Dario; Barbon, Elena; Scalet, Daniela; Cavallari, Nicola; Perrone, Daniela; Zanibellato..., Silvia; Bernardi, Francesco; Pinotti, Mirko | |
| An exon-specific U1snRNA induces a robust factor IX activity in mice expressing multiple human FIX splicing mutants | 2016 | Balestra, Dario; Scalet, Daniela; Pagani, Franco; Rogalska, Malgorzata Ewa; Mari, Rosella; Bernar...di, Francesco; Pinotti, Mirko | |
| Regulation of exon definition by intrinsic elements and by combination of tailored U1snRNA with antisense oligonucleotides | 2016 | Scalet, Daniela | |
| Exploring Splicing-Switching Molecules For Seckel Syndrome Therapy | 2017 | Scalet, Daniela; Balestra, Dario; Rohban, Sara; Bovolenta, Matteo; Perrone, Daniela; Bernardi, Fr...ancesco; Campaner, Stefano; Pinotti, Mirko | |
| Rescue of missense and splicing mutations in Haemophilia A by a unique Exon Specific U1snRNA | 2018 | Balestra, Dario; Scalet, Daniela; Leo, Gabriele; Donadon, I.; Lombardi, Silvia; Bernardi, Frances...co; Pinotti, M. | |
| The somatic FAH C.1061C>A change counteracts the frequent FAH c.1062+5G>A mutation and permits U1snRNA-based splicing correction | 2018 | Scalet, Daniela; Sacchetto, Claudia; Bernardi, Francesco; Pinotti, Mirko; Van De Graaf, Stan F. J....; Balestra, Dario | |
| Disease-causing variants of the conserved+2T of 5 ' splice sites can be rescued by engineered U1snRNAs | 2019 | Scalet, Daniela; Maestri, Iva; Branchini, Alessio; Bernardi, Francesco; Pinotti, Mirko; Balestra,... Dario | |
| A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I | 2020 | 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. |
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