LOMBARDI, Silvia

LOMBARDI, Silvia  

Dipartimento di Scienze della vita e biotecnologie  

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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
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
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
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.
Deep molecular mechanisms of F8 exon 19 variants and translational approaches in Hemophilia A 2021 Lombardi, Silvia; Peretto, Laura; Merlin, Simone; Follenzi, Antonia; Mcvey, John H.; Maestri, Iva...; Bernardi, Francesco; Pinotti, Mirko; Balestra, Dario file con accesso da definire
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
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
Detection of Residual Factor VIII Levels Reveals the Occurrence of Readthrough Over the Majority of F8 Nonsense Mutations 2020 Testa, Maria Francesca; Lombardi, Silvia; Ferrarese, Mattia; Pinotti, Mirko; Castaman, Giancarlo;... Radossi, Paolo; Belvini, Donata; Bernardi, Francesco; Branchini, Alessio
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
Dissection of pleiotropic effects of variants in and adjacent to F8 exon 19 and rescue of mRNA splicing and protein function 2021 Lombardi, S.; Leo, G.; Merlin, S.; Follenzi, A.; Mcvey, J. H.; Maestri, I.; Bernardi, F.; Pinotti..., M.; Balestra, D. file con accesso da definire
An engineered human albumin enhances half-life and transmucosal delivery when fused to protein-based biologics 2020 Bern, M.; Nilsen, J.; Ferrarese, M.; Sand, K. M. K.; Gjolberg, T. T.; Lode, H. E.; Davidson, R. J....; Camire, R. M.; Baekkevold, E. S.; Foss, S.; Grevys, A.; Dalhus, B.; Wilson, J.; Hoydahl, L. S.; Christianson, G. J.; Roopenian, D. C.; Schlothauer, T.; Michaelsen, T. E.; Moe, M. C.; Lombardi, S.; Pinotti, M.; Sandlie, I.; Branchini, A.; Andersen, J. T.
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.
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
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
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 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
Fusion of engineered albumin with factor IX Padua extends half-life and improves coagulant activity 2021 Lombardi, Silvia; Aaen, Kristin H; Nilsen, Jeannette; Ferrarese, Mattia; Gjølberg, Torleif T; Ber...nardi, Francesco; Pinotti, Mirko; Andersen, Jan T; Branchini, Alessio
Molecular insights into determinants of translational readthrough and implications for nonsense suppression approaches 2020 Lombardi, S.; Testa, M. F.; Pinotti, M.; Branchini, A.
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