Sfoglia per Autore
Responsiveness of hemophilia B- causing non sense mutations to ribosome readthrough-inducing drugs strictly depends on the nucleotide and prrotein context
file con accesso da definire2016 Branchini, Alessio; Ferrarese, Mattia; Lombardi, Silvia; Baroni, Marcello; Campioni, Matteo; Burini, Francesco; Bernardi, Francesco; Pinotti, Mirko
Differential functional readthrough over homozygous nonsense mutations contributes to the bleeding phenotype in coagulation factor VII deficiency
2016 Branchini, Alessio; Ferrarese, Mattia; Lombardi, Silvia; Mari, Rosella; Bernardi, Francesco; Pinotti, Mirko
Exploring chaperone-like compounds as innovative therapeutic strategy for Hemophilia B
file con accesso da definire2017 Pignani, S; Ferrarese, M; Lombardi, S; Marchi, S; Todaro, A; Pinton, P; Bernardi, F; Pinotti, M; Branchini, A
Exploring chaperone-like compounds as innovative therapeutic correction approach for factor IX missense mutations causing type I Haemophilia B
2017 Pignani, S; Todaro, A; Ferrarese, M; Marchi, S; Lombardi, S; Pinton, P; Bernardi, F; Pinotti, M; Branchini, A
The carboxyl-terminal region of human coagulation factor X as a novel naturally-occuring linker for fusion strategies
2018 Ferrarese, Mattia; Pignani, Silvia; Lombardi, Silvia; Balestra, Dario; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
The chaperone-like compound sodium phenylbutyrate improves intracellular trafficking, secretion and coagulant activity of factor IX impaired by the frequent p.R294Q mutation
2018 Pignani, Silvia; Todaro, Alice; Ferrarese, Mattia; Marchi, Saverio; Lombardi, Silvia; Balestra, Dario; Pinton, Paolo; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
A strategy with chaperone-like compounds to restore expression of factor IX variants affected by frequent missense mutations causing hemophilia B
2018 Pignani, Silvia; Todaro, Alice; Ferrarese, Mattia; Marchi, Saverio; Lombardi, Silvia; Balestra, Dario; Pinton, Paolo; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
The chaperone-like sodium phenylbutyrate improves factor IX intracellular trafficking and activity impaired by the frequent p.R294Q mutation
2018 Pignani, S; Todaro, A; Ferrarese, M; Marchi, S; Lombardi, S; Balestra, D; Pinton, P; Bernardi, F; Pinotti, M; Branchini, A
Readthrough-mediated functional suppression of homozygous nonsense mutations accounts for variable bleeding phenotypes in factor VII deficiency
file con accesso da definire2018 Lombardi, Silvia; Ferrarese, Mattia; Pinotti, Mirko; Mari, Rosella; Bernardi, Francesco; Branchini, Alessio
Rescue of multiple Haemophilia A-causing mutations by a single ExSpeU1: the importance of the genomic context
2019 Balestra, Dario; Lombardi, Silvia; Leo, Gabriele; Calanchi, Irene; Bernardi, Francesco; Pinotti, Mirko
Exploring spontaneous readthrough over recurrent F8 nonsense mutations: potential correlation with inhibitor risk?
2019 Lombardi, Silvia; Testa, Maria Francesca; Pinotti, Mirko; Castman, Giancarlo; Radossi, Paolo; Bernardi, Francesco; Branchini, Alessio
Exon-Specific U1snRNA-Mediated Rescue of Splicing and Missense Changes in Hemophilia A
2019 Lombardi, Silvia; Leo, Gabriele; Maestri, Iva; Bernardi, Francesco; Pinotti, Mirko; Mcvey, John Henderson; Balestra, Dario
The carboxyl-terminal region of human coagulation factor X as a natural linker for fusion strategies
2019 Ferrarese, Mattia; Pignani, Silvia; Lombardi, Silvia; Balestra, Dario; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
Design of a novel factor IX variant with enhanced procoagulant activity and half-life
2020 Branchini, Alessio; Lombardi, Silvia; Nilsen, Jeannette; Hovden Aaen, Kristin; Ferrarese, Mattia; Bern, Malin; Roopenian, Derry C.; Sandlie, Inger; Terje Andersen, Jan; Pinotti, Mirko
A next-generation rFVIIa fusion protein with enhanced half-life as a novel by-passing tool in hemophilia
2020 Branchini, Alessio; Ferrarese, Mattia; Nilsen, Jeannette; Bern, Malin; Davidson, Robert J.; Camire, Rodney M.; Roopenian, Derry C.; Sandlie, Inger; Lombardi, Silvia; Terje Andersen, Jan; Pinotti, Mirko
Rational engineering of a novel factor IX albumin fusion protein results in enhanced coagulant activity and pharmacokinetic profile
2020 Lombardi, Silvia; Nilsen, Jeannette; Hovden Aaen, Kristin; Ferrarese, Mattia; Pinotti, Mirko; Bern, Malin; Roopenian, Derry C.; Sandlie, Inger; Terje Andersen, Jan; Branchini, Alessio
Spontaneous readthrough over recurrent F8 nonsense mutations is associated with residual factor VIII levels: implications for inhibitor risk?
2020 Testa, Maria Francesca; Lombardi, Silvia; Ferrarese, Mattia; Pinotti, Mirko; Castaman, Giancarlo; Radossi, Paolo; Belvini, Donata; Bernardi, Francesco; Branchini, Alessio
Design of a novel factor IX albumin fusion protein with enhanced coagulant activity and pharmacokinetic profile
2020 Lombardi, Silvia; Nilsen, Jeannette; Hovden Aaen, Kristin; Ferrarese, Mattia; Pinotti, Mirko; Bern, Malin; Roopenian, Derry C.; Sandlie, Inger; Terje Andersen, Jan; Branchini, Alessio
An exon-specific small nuclear u1 rna (Exspeu1) improves hepatic otc expression in a splicing-defective spf /ash mouse model of ornithine transcarbamylase deficiency
2020 Balestra, D.; Ferrarese, M.; Lombardi, S.; Ziliotto, N.; Branchini, A.; Petersen, N.; Bosma, P.; Pinotti, M.; van de Graaf, S. F. J.
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 |
|---|---|---|---|
| Responsiveness of hemophilia B- causing non sense mutations to ribosome readthrough-inducing drugs strictly depends on the nucleotide and prrotein context | 2016 | Branchini, Alessio; Ferrarese, Mattia; Lombardi, Silvia; Baroni, Marcello; Campioni, Matteo; Buri...ni, Francesco; Bernardi, Francesco; Pinotti, Mirko | file con accesso da definire |
| Differential functional readthrough over homozygous nonsense mutations contributes to the bleeding phenotype in coagulation factor VII deficiency | 2016 | Branchini, Alessio; Ferrarese, Mattia; Lombardi, Silvia; Mari, Rosella; Bernardi, Francesco; Pino...tti, Mirko | |
| Exploring chaperone-like compounds as innovative therapeutic strategy for Hemophilia B | 2017 | Pignani, S; Ferrarese, M; Lombardi, S; Marchi, S; Todaro, A; Pinton, P; Bernardi, F; Pinotti, M; ...Branchini, A | file con accesso da definire |
| Exploring chaperone-like compounds as innovative therapeutic correction approach for factor IX missense mutations causing type I Haemophilia B | 2017 | Pignani, S; Todaro, A; Ferrarese, M; Marchi, S; Lombardi, S; Pinton, P; Bernardi, F; Pinotti, M; ...Branchini, A | |
| The carboxyl-terminal region of human coagulation factor X as a novel naturally-occuring linker for fusion strategies | 2018 | Ferrarese, Mattia; Pignani, Silvia; Lombardi, Silvia; Balestra, Dario; Bernardi, Francesco; Pinot...ti, Mirko; Branchini, Alessio | |
| The chaperone-like compound sodium phenylbutyrate improves intracellular trafficking, secretion and coagulant activity of factor IX impaired by the frequent p.R294Q mutation | 2018 | Pignani, Silvia; Todaro, Alice; Ferrarese, Mattia; Marchi, Saverio; Lombardi, Silvia; Balestra, D...ario; Pinton, Paolo; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio | |
| A strategy with chaperone-like compounds to restore expression of factor IX variants affected by frequent missense mutations causing hemophilia B | 2018 | Pignani, Silvia; Todaro, Alice; Ferrarese, Mattia; Marchi, Saverio; Lombardi, Silvia; Balestra, D...ario; Pinton, Paolo; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio | |
| The chaperone-like sodium phenylbutyrate improves factor IX intracellular trafficking and activity impaired by the frequent p.R294Q mutation | 2018 | Pignani, S; Todaro, A; Ferrarese, M; Marchi, S; Lombardi, S; Balestra, D; Pinton, P; Bernardi, F;... Pinotti, M; Branchini, A | |
| Readthrough-mediated functional suppression of homozygous nonsense mutations accounts for variable bleeding phenotypes in factor VII deficiency | 2018 | Lombardi, Silvia; Ferrarese, Mattia; Pinotti, Mirko; Mari, Rosella; Bernardi, Francesco; Branchin...i, Alessio | file con accesso da definire |
| Rescue of multiple Haemophilia A-causing mutations by a single ExSpeU1: the importance of the genomic context | 2019 | Balestra, Dario; Lombardi, Silvia; Leo, Gabriele; Calanchi, Irene; Bernardi, Francesco; Pinotti, ...Mirko | |
| Exploring spontaneous readthrough over recurrent F8 nonsense mutations: potential correlation with inhibitor risk? | 2019 | Lombardi, Silvia; Testa, Maria Francesca; Pinotti, Mirko; Castman, Giancarlo; Radossi, Paolo; Ber...nardi, Francesco; Branchini, Alessio | |
| Exon-Specific U1snRNA-Mediated Rescue of Splicing and Missense Changes in Hemophilia A | 2019 | Lombardi, Silvia; Leo, Gabriele; Maestri, Iva; Bernardi, Francesco; Pinotti, Mirko; Mcvey, John H...enderson; Balestra, Dario | |
| The carboxyl-terminal region of human coagulation factor X as a natural linker for fusion strategies | 2019 | Ferrarese, Mattia; Pignani, Silvia; Lombardi, Silvia; Balestra, Dario; Bernardi, Francesco; Pinot...ti, Mirko; Branchini, Alessio | |
| Design of a novel factor IX variant with enhanced procoagulant activity and half-life | 2020 | Branchini, Alessio; Lombardi, Silvia; Nilsen, Jeannette; Hovden Aaen, Kristin; Ferrarese, Mattia;... Bern, Malin; Roopenian, Derry C.; Sandlie, Inger; Terje Andersen, Jan; Pinotti, Mirko | |
| A next-generation rFVIIa fusion protein with enhanced half-life as a novel by-passing tool in hemophilia | 2020 | Branchini, Alessio; Ferrarese, Mattia; Nilsen, Jeannette; Bern, Malin; Davidson, Robert J.; Camir...e, Rodney M.; Roopenian, Derry C.; Sandlie, Inger; Lombardi, Silvia; Terje Andersen, Jan; Pinotti, Mirko | |
| Rational engineering of a novel factor IX albumin fusion protein results in enhanced coagulant activity and pharmacokinetic profile | 2020 | Lombardi, Silvia; Nilsen, Jeannette; Hovden Aaen, Kristin; Ferrarese, Mattia; Pinotti, Mirko; Ber...n, Malin; Roopenian, Derry C.; Sandlie, Inger; Terje Andersen, Jan; Branchini, Alessio | |
| Spontaneous readthrough over recurrent F8 nonsense mutations is associated with residual factor VIII levels: implications for inhibitor risk? | 2020 | Testa, Maria Francesca; Lombardi, Silvia; Ferrarese, Mattia; Pinotti, Mirko; Castaman, Giancarlo;... Radossi, Paolo; Belvini, Donata; Bernardi, Francesco; Branchini, Alessio | |
| Design of a novel factor IX albumin fusion protein with enhanced coagulant activity and pharmacokinetic profile | 2020 | Lombardi, Silvia; Nilsen, Jeannette; Hovden Aaen, Kristin; Ferrarese, Mattia; Pinotti, Mirko; Ber...n, Malin; Roopenian, Derry C.; Sandlie, Inger; Terje Andersen, Jan; Branchini, Alessio | |
| An exon-specific small nuclear u1 rna (Exspeu1) improves hepatic otc expression in a splicing-defective spf /ash mouse model of ornithine transcarbamylase deficiency | 2020 | Balestra, D.; Ferrarese, M.; Lombardi, S.; Ziliotto, N.; Branchini, A.; Petersen, N.; Bosma, P.; ...Pinotti, M.; van de Graaf, S. F. J. | |
| 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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