FERRARESE, Mattia

FERRARESE, Mattia  

Dipartimento di Scienze della vita e biotecnologie  

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Titolo Data di pubblicazione Autore(i) File
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.
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
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
Akt-mediated phosphorylation of MICU1 regulates mitochondrial Ca 2+ levels and tumor growth 2019 Marchi, Saverio; Corricelli, Mariangela; Branchini, Alessio; Vitto, VERONICA ANGELA MARIA; Missir...oli, Sonia; Morciano, Giampaolo; Perrone, Mariasole; Ferrarese, Mattia; Giorgi, Carlotta; Pinotti, Mirko; Galluzzi, Lorenzo; Kroemer, Guido; Pinton, Paolo
An advanced method for the small-scale production of high-quality minicircle DNA 2021 Mitdank, Hardy; Sama, Simko; Tröger, Meike; Testa, Maria Francesca; Ferrarese, Mattia; Balestra, ...Dario; Pinotti, Mirko; Weng, Alexander
An Altered Splicing Registry Explains the Differential ExSpeU1-Mediated Rescue of Splicing Mutations Causing Haemophilia A 2019 Balestra, Dario; Maestri, Iva; Branchini, Alessio; Ferrarese, Mattia; Bernardi, Francesco; Pinott...i, Mirko
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.
An optimized in vitro expression platform identifies Haemophilia B nonsense mutations, and thus patients, eligible for therapeutic drug-induced readthrough 2017 Branchini, Alessio; Ferrarese, Mattia; Castaman, Giancarlo; Bernardi, Francesco; Pinotti, Mirko
Comparative Analysis Of Residual Factor VIII Expression from Recurrent F8 Nonsense Mutations Indicates that Localization in the B- domain Favours Readthrough- mediated Protein Output 2021 Testa, Maria Francesca; Lombardi, Silvia; Ferrarese, Mattia; Radossi, Paolo; Belvini, Donata; Cas...taman, Giancarlo; Bernardi, Francesco; Pinotti, Mirko; 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
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
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
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
Identification of novel mechanisms underlying functional response to drug-induced readthrough of haemophilia B nonsense mutations 2018 Ferrarese, Mattia; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio
Missense changes in the catalytic domain of coagulation factor X account for minimal function preventing a perinatal lethal condition 2019 Ferrarese, Mattia; Baroni, Marcello; Della Valle, Patrizia; Spiga, Ivana; Poloniato, Antonella; D...'Angelo, Armando; Pinotti, Mirko; Bernardi, Francesco; Branchini, Alessio
Mutation-specific contributions to trace factor X levels account for a life-threating phenotype in a compound heterozygous factor X deficient patient 2018 Ferrarese, Mattia; Baroni, Marcello; Della Valle, Patrizia; Spiga, Ivana; Poloniato, Antonella; D...'Angelo, Armando; Bernardi, Francesco; Branchini, Alessio file con accesso da definire