| The chaperone‐like sodium phenylbutyrate improves factor IX intracellular trafficking and activity impaired by the frequent p. R294Q mutation S Pignani, A Todaro, M Ferrarese, S Marchi, S Lombardi, D Balestra, ... Journal of Thrombosis and Haemostasis 16 (10), 2035-2043, 2018 | 18 | 2018 |
| An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes E Barbon, S Pignani, A Branchini, F Bernardi, M Pinotti, M Bovolenta Scientific reports 6 (1), 28304, 2016 | 18 | 2016 |
| Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts S Pignani, F Zappaterra, E Barbon, A Follenzi, M Bovolenta, F Bernardi, ... Biochimica et Biophysica Acta (BBA)-Gene Regulatory Mechanisms 1862 (6), 619-624, 2019 | 8 | 2019 |
| The carboxyl-terminal region of human coagulation factor X as a natural linker for fusion strategies M Ferrarese, S Pignani, S Lombardi, D Balestra, F Bernardi, M Pinotti, ... Thrombosis Research 173, 4-11, 2019 | 7 | 2019 |
| Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy R Famà, E Borroni, S Merlin, C Airoldi, S Pignani, A Cucci, D Corà, ... haematologica 106 (6), 1624, 2021 | 6 | 2021 |
| Modulation of F8 promoter activity in response to Ets-1 and Ets-2 transcription factors for hemophilia A gene therapy application R Fama, E Borroni, S Merlin, S Pignani, A Cucci, S Scardellato, A Follenzi HUMAN GENE THERAPY 30 (11), A140-A140, 2019 | | 2019 |
| CRISPR activation on coagulation F7 or F8 promoters potentiate trascriptional activity in the normal and mutated gene context S Pignani, F Zappaterra, E Barbon, M Bovolenta, F Bernardi, A Branchini, ... RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS 3 (S1), 105-106, 2019 | | 2019 |
| Modulation at transcriptional and post-translational level to develop therapeutic approaches for coagulation factor deficiencies S Pignani Università degli studi di Ferrara, 2018 | | 2018 |
| The chaperone-like compound sodium phenylbutyrate improves intracellular trafficking, secretion and coagulant activity of factor IX impaired by the frequent p. R294Q mutation S Pignani, A Todaro, M Ferrarese, S Marchi, S Lombardi, D Balestra, ... BLOOD TRANSFUSION 16 (Supplement 4), 418-418, 2018 | | 2018 |
| The carboxyl-terminal region of human coagulation factor X as a novel naturally-occuring linker for fusion strategies M Ferrarese, S Pignani, S Lombardi, D Balestra, F Bernardi, M Pinotti, ... BLOOD TRANSFUSION 16 (Supplement 4), 418-418, 2018 | | 2018 |
| A strategy with chaperone-like compounds to restore expression of factor IX variants affected by frequent missense mutations causing hemophilia B S Pignani, A Todaro, M Ferrarese, S Marchi, S Lombardi, D Balestra, ... HEMASPHERE 2 (S1), 393-393, 2018 | | 2018 |
| Exploring chaperone-like compounds as innovative therapeutic correction approach for factor IX missense mutations causing type I Haemophilia B S Pignani, A Todaro, M Ferrarese, S Marchi, S Lombardi, P Pinton, ... BLOOD TRANSFUSION 15 (Suppl 4), 541-541, 2017 | | 2017 |
| Exploring chaperone-like compounds as innovative therapeutic strategy for Hemophilia B S Pignani, M Ferrarese, S Lombardi, S Marchi, A Todaro, P Pinton, ... RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS 1 (S1), 155-155, 2017 | | 2017 |
| Engineered transcription factors (TALE-TF) as potential therapeutic strategy for coagulation factor deficiencies caused by promoter mutations E Barbon, S Pignani, A Branchini, F Bernardi, M Pinotti, M Bovolenta JOURNAL OF THROMBOSIS AND HAEMOSTASIS 13 (S2), 162-163, 2015 | | 2015 |
