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Recombinant Expression of F9 Nonsense Mutations and Fix Pharmacokinetics in Hemophilia B
Branchini, Alessio; Ferrarese, Mattia; Pinotti, Mirko; Bernardi, Francesco; Morfini, Massimo    
Wiley, Abstracts of the 64th Annual Meeting of the Scientific Standardization Committee of the International Society on Thrombosis and Haemostasis
Vol. 2, No. S1, pp: 66-66, Anno: 2018

Favourable recombinant factor IX pharmacokinetics outcomes in severe hemophilia B patients with FIX activation site mutations
Branchini, A.; Morfini, M.; Zordan, T.; Bonsi, M.; Pinotti, M.; Serino, M. L.; Radossi, P.; Belvini, D.; Castaman, G.; Bernardi, F.     dettagli >>
Wiley, Abstracts of the XXVII Congress of the International Society on Thrombosis and Haemostasis, July 6-10, 2019
Vol. 3, No. S1, pp: 250-250, Anno: 2019

Next generation factor VIIa with enhanced half-life
Ferrarese, M.; Nilsen, J.; Pinotti, M.; Bern, M.; Davidson, R. J.; Camire, R. M.; Lode, H. E.; Roopenian, D. C.; Sandlie, I.; Lombardi, S.; Castaman, G.; Andersen, J. T.; Branchini, A.     dettagli >>
Wiley, Abstracts of the XXVII Congress of the International Society on Thrombosis and Haemostasis, July 6-10, 2019
Vol. 3, No. S1, pp: 312-313, Anno: 2019

CRISPR activation on coagulation F7 or F8 promoters potentiate trascriptional activity in the normal and mutated gene context
Pignani, Silvia; Zappaterra, Federico; Barbon, Elena; Bovolenta, Matteo; Bernardi, Francesco; Branchini, Alessio; Pinotti, Mirko     dettagli >>
Wiley, RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
Vol. 3, No. S1, pp: 105-106, Anno: 2019

Exploring spontaneous readthrough over recurrent F8 nonsense mutations: potential correlation with inhibitor risk?
Lombardi, Silvia; Testa, Maria Francesca; Pinotti, Mirko; Castman, Giancarlo; Radossi, Paolo; Bernardi, Francesco; Branchini, Alessio     dettagli >>
Wiley, RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
Vol. 3, No. S1, pp: 110-111, Anno: 2019

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 >>
Edizioni SIMTI, Blood transfusion
Vol. 16, No. 4, pp: 454-454, Anno: 2018

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 >>
Walter Ageno, Anna Falanga, Gianccarlo Castaman, BLOOD TRANSFUSION
Vol. 16, No. Supplement 4, pp: 418-418, Anno: 2018

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 >>
Walter Ageno, Anna Falanga, Gianccarlo Castaman, BLOOD TRANSFUSION
Vol. 16, No. Supplement 4, pp: 456-456, Anno: 2018

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 >>
Walter Ageno, Anna Falanga, Gianccarlo Castaman, BLOOD TRANSFUSION
Vol. 16, No. Supplement 4, pp: 418-418, Anno: 2018

Readthrough-mediated functional suppression of homozygous nonsense mutations accounts for variable bleeding phenotypes in factor VII deficiency
Lombardi, Silvia; Ferrarese, Mattia; Pinotti, Mirko; Mari, Rosella; Bernardi, Francesco; Branchini, Alessio     dettagli >>
Walter Ageno, Anna Falanga, Gianccarlo Castaman, BLOOD TRANSFUSION
Vol. 16, No. Supplement 4453, pp: 453-454, Anno: 2018

The carboxyl-terminal region is not essential for secreted and functional levels of coagulation factor X
Branchini, A.; Baroni, M.; Burini, F.; Mari, R.; Gemmati, D.; Puzzo, F.; Bernardi, F.; Pinotti, M.     dettagli >>
Anna Falanga, BLOOD TRANSFUSION
Vol. 14, No. Suppl 5, pp: 717-717, Anno: 2016

MOLECULAR MECHANISMS AND THERAPEUTIC APROACHES FOR RESTORATION OF mRNA TRANSCRIPTION, MATURATION AND TRANSLATION IN INHERITED COAGULATION FACTOR DEFICIENCIES
Balestra, D; Bovolenta, M; Branchini, A; Pinotti, M; Bernardi, F     dettagli >>
Anna Falanga, BLOOD TRANSFUSION
Vol. 14, No. 1, pp: 694-706, Anno: 2016

RESPONSIVENESS OF HEMOPHILIA B-CAUSING NONSENSE MUTATIONS TO RIBOSOME READTHROUGH-INDUCING DRUGS STRICTLY DEPENDS ON THE NUCLEOTIDE AND PROTEIN CONTEXT
Branchini, Alessio; Ferrarese, Mattia; Lombardi, Silvia; Baroni, Marcello; Campioni, Matteo; Burini, Francesco; Bernardi, Francesco; Pinotti, Mirko     dettagli >>
Ferrata-Storti Foundation/European Hematology Association, HAEMATOLOGICA
Vol. 101, No. s1, pp: 169-170, Anno: 2016

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 >>
Wolters Kluwer, Abstract book of the 23rd Congress of the European Hematology Association
Vol. 2, No. S1, pp: 393-393, Anno: 2018

Identification of novel mechanisms underlying functional response to drug-induced readthrough of haemophilia B nonsense mutations
Ferrarese, Mattia; Bernardi, Francesco; Pinotti, Mirko; Branchini, Alessio    
Wolters Kluwer, Abstract book of the 23rd Congress of the European Hematology Association
Vol. 2, No. S1, pp: 99-99, Anno: 2018

An optimized in vitro expression platform identifies Haemophilia B nonsense mutations, and thus patients, eligible for therapeutic drug-induced readthrough
Branchini, Alessio; Ferrarese, Mattia; Castaman, Giancarlo; Bernardi, Francesco; Pinotti, Mirko    
Edizioni SIMTI, Blood Transfusion
Vol. 15, No. Suppl 4, pp: 540-540, Anno: 2017

Exploring chaperone-like compounds as innovative therapeutic strategy for Hemophilia B
Pignani, S; Ferrarese, M; Lombardi, S; Marchi, S; Todaro, A; Pinton, P; Bernardi, F; Pinotti, M; Branchini, A    
Wiley, Abstracts of the XXVI Congress of the International Society on Thrombosis and Haemostasis, July 8-13, 2017
Vol. 1, No. S1, pp: 155-155, Anno: 2017

Exploring chaperone-like compounds as innovative therapeutic correction approach for factor IX missense mutations causing type I Haemophilia B
Pignani, Silvia; Todaro, A; Ferrarese, M; Marchi, S; Lombardi, S; Pinton, P; Bernardi, F; Pinotti, M; Branchini, A    
Edizioni SIMTI, Blood Transfusion
Vol. 15, No. Suppl 4, pp: 541-541, Anno: 2017

A unique exon specific U1snRNA rescues different haemophilia B - causing splicing-defective factor IX variants in mice
Balestra, D; Scalet, D; Pagani, F; Bernardi, F; Pinotti, M    
WILEY-BLACKWELL, JOURNAL OF THROMBOSIS AND HAEMOSTASIS
pp: 208-209, Anno: 2015

Correction of aberrant splicing causing haemophilia B through the combination of compensatory U1snRNAs and antisense oligonucleotides
Scalet, D; Balestra, D; Barbon, E; Cavallari, N; Perrone, D; Bernardi, F; Pinotti, M    
WILEY-BLACKWELL, JOURNAL OF THROMBOSIS AND HAEMOSTASIS
pp: 210-210, Anno: 2015

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