Sfoglia per Autore
Engineered transcription factors (TALE-TF) as potential therapeutic strategy for coagulation factor deficiencies caused by promoter mutations
file con accesso da definire2015 Barbon, E; Pignani, S; Branchini, A; Bernardi, F; Pinotti, M; Bovolenta, M
An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes
2016 Barbon, Elena; Pignani, Silvia; Branchini, Alessio; Bernardi, Francesco; Pinotti, Mirko; Bovolenta, Matteo
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
Modulation at transcriptional and post-translational level to develop therapeutic approaches for coagulation factor deficiencies
2018 Pignani, Silvia
CRISPR activation on coagulation F7 or F8 promoters potentiate trascriptional activity in the normal and mutated gene context
2019 Pignani, Silvia; Zappaterra, Federico; Barbon, Elena; Bovolenta, Matteo; Bernardi, Francesco; Branchini, Alessio; Pinotti, Mirko
Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts
2019 Pignani, Silvia; Zappaterra, Federico; Barbon, Elena; Follenzi, Antonia; Bovolenta, Matteo; Bernardi, Francesco; Branchini, Alessio; Pinotti, Mirko
Base and Prime editing of DNA as a new therapeutic option for Hemophilia A
file con accesso da definire2022 Tonetto, Elena; Pignani, Silvia; Roman, Giacomo; Follenzi, Antonia; Bernardi, Francesco; Liu, David; Pinotti, Mirko; Balestra, Dario
New Genome Editing Approaches: Base And Prime Editing To Revert Hemophilia A-Causing Point Mutations
file con accesso da definire2022 Tonetto, E.; Pignani, S.; Roman, G.; Follenzi, A.; Bernardi, F.; Liu, D.; Pinotti, M.; Balestra, D.
| Titolo | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|
| Engineered transcription factors (TALE-TF) as potential therapeutic strategy for coagulation factor deficiencies caused by promoter mutations | 2015 | Barbon, E; Pignani, S; Branchini, A; Bernardi, F; Pinotti, M; Bovolenta, M | file con accesso da definire |
| An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes | 2016 | Barbon, Elena; Pignani, Silvia; Branchini, Alessio; Bernardi, Francesco; Pinotti, Mirko; Bovolent...a, Matteo | |
| 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 | |
| Modulation at transcriptional and post-translational level to develop therapeutic approaches for coagulation factor deficiencies | 2018 | Pignani, Silvia | |
| CRISPR activation on coagulation F7 or F8 promoters potentiate trascriptional activity in the normal and mutated gene context | 2019 | Pignani, Silvia; Zappaterra, Federico; Barbon, Elena; Bovolenta, Matteo; Bernardi, Francesco; Bra...nchini, Alessio; Pinotti, Mirko | |
| Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts | 2019 | Pignani, Silvia; Zappaterra, Federico; Barbon, Elena; Follenzi, Antonia; Bovolenta, Matteo; Berna...rdi, Francesco; Branchini, Alessio; Pinotti, Mirko | |
| Base and Prime editing of DNA as a new therapeutic option for Hemophilia A | 2022 | Tonetto, Elena; Pignani, Silvia; Roman, Giacomo; Follenzi, Antonia; Bernardi, Francesco; Liu, Dav...id; Pinotti, Mirko; Balestra, Dario | file con accesso da definire |
| New Genome Editing Approaches: Base And Prime Editing To Revert Hemophilia A-Causing Point Mutations | 2022 | Tonetto, E.; Pignani, S.; Roman, G.; Follenzi, A.; Bernardi, F.; Liu, D.; Pinotti, M.; Balestra, D. | file con accesso da definire |
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