BALESTRA, Dario
BALESTRA, Dario
Dipartimento di Scienze della vita e biotecnologie
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 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
A unique exon specific U1snRNA rescues different haemophilia B - causing splicing-defective factor IX variants in mice
file con accesso da definire2015 Balestra, Dario; Scalet, Daniela; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko
A very rare simultaneous presence of a ring chromosome 13 and a splicing site mutation on Factor X gene
file con accesso da definire2013 M., Menegatti; Balestra, Dario; B., Fabrizzi; R., Asselta; Pinotti, Mirko; F., Peyvandi
Aberrant splicing reverts a potentially lethal coagulation deficiency caused by a +1g/t splicing mutation
file con accesso da definire2011 Cavallari, Nicola; Balestra, Dario; Rizzotto, Lara; Maestri, Iva; A., Chamsunri; Bernardi, Francesco; Pinotti, Mirko
Activation of a cryptic splice site in a potentially lethal coagulation defect accounts for a functional protein variant
file con accesso da definire2012 Cavallari, Nicola; Balestra, Dario; Branchini, Alessio; Maestri, Iva; Chuamsunrit, A; Sasanakul, W; Mariani, G; Pagani, F; Bernardi, Francesco; Pinotti, Mirko
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; Pinotti, Mirko
An engineered U1 small nuclear RNA rescues splicing-defective coagulationF7gene expression in mice
file con accesso da definire2014 Balestra, Dario; A., Faella; P., Margaritis; N., Cavallari; F., Pagani; Bernardi, Francesco; V. R., Arruda; Pinotti, Mirko
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 exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects
file con accesso da definire2012 E., Fernandez Alanis; Pinotti, Mirko; A., Dal Mas; Balestra, Dario; Cavallari, Nicola; Me, Rogalska; Bernardi, Francesco; F., Pagani
AN EXON-SPECIFIC U1 SMALL NUCLEAR RNA (snRNA) STRATEGY TO CORRECT SPLICING MUTATIONS ASSOCIATED TO HEMOPHILIA B
file con accesso da definire2012 Balestra, Dario; Cavallari, Nicola; E., Fernandez Alanis; A., Dal Mas; M. E., Rogalska; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko
An exon-specific U1snRNA induces a robust factor IX activity in mice expressing multiple human FIX splicing mutants
2016 Balestra, Dario; Scalet, Daniela; Pagani, Franco; Rogalska, Malgorzata Ewa; Mari, Rosella; Bernardi, Francesco; 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
Base and Prime Editors as new correction approaches for recurrent Hemophilia A Nonsense Mutations
file con accesso da definire2024 Testa, M.; Bernardi, F.; Branchini, A.; Pinotti, M.; Balestra, D.
C6orf10 low-frequency and rare variants in italian multiple sclerosis patients
2019 Ziliotto, Nicole; Marchetti, Giovanna; Scapoli, Chiara; Bovolenta, Matteo; Meneghetti, Silvia; Benazzo, Andrea; Lunghi, Barbara; Balestra, Dario; Laino, Lorenza Anna; Bozzini, Nicolò; Guidi, Irene; Salvi, Fabrizio; Straudi, Sofia; Gemmati, Donato; Menegatti, Erica; Zamboni, Paolo; Bernardi, Francesco
Contribution of asialoglycoprotein receptor ASGR2 5’ UTR polymorphisms to full-length FVIII concentrate pharmacokinetics
2020 Lunghi, B.; Morfini, M.; Martinelli, N.; Frusconi, S.; Balestra, D.; Branchini, A.; Linari, S.; Marchetti, G.; Castaman, G.; Bernardi, F.
Correction of aberrant splicing causing haemophilia B through the combination of compensatory U1snRNAs and antisense oligonucleotides
file con accesso da definire2015 Scalet, Daniela; Balestra, Dario; Barbon, Elena; Cavallari, Nicola; Perrone, Daniela; Bernardi, Francesco; Pinotti, Mirko
Counteracting the Common Shwachman-Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing
2023 Peretto, Laura; Tonetto, Elena; Maestri, Iva; Bezzerri, Valentino; Valli, Roberto; Cipolli, Marco; Pinotti, Mirko; Balestra, Dario
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
| 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 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 | |
| A unique exon specific U1snRNA rescues different haemophilia B - causing splicing-defective factor IX variants in mice | 2015 | Balestra, Dario; Scalet, Daniela; Pagani, Franco; Bernardi, Francesco; Pinotti, Mirko | file con accesso da definire |
| A very rare simultaneous presence of a ring chromosome 13 and a splicing site mutation on Factor X gene | 2013 | M., Menegatti; Balestra, Dario; B., Fabrizzi; R., Asselta; Pinotti, Mirko; F., Peyvandi | file con accesso da definire |
| Aberrant splicing reverts a potentially lethal coagulation deficiency caused by a +1g/t splicing mutation | 2011 | Cavallari, Nicola; Balestra, Dario; Rizzotto, Lara; Maestri, Iva; A., Chamsunri; Bernardi, France...sco; Pinotti, Mirko | file con accesso da definire |
| Activation of a cryptic splice site in a potentially lethal coagulation defect accounts for a functional protein variant | 2012 | Cavallari, Nicola; Balestra, Dario; Branchini, Alessio; Maestri, Iva; Chuamsunrit, A; Sasanakul, ...W; Mariani, G; Pagani, F; Bernardi, Francesco; Pinotti, Mirko | file con accesso da definire |
| 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 U1 small nuclear RNA rescues splicing-defective coagulationF7gene expression in mice | 2014 | Balestra, Dario; A., Faella; P., Margaritis; N., Cavallari; F., Pagani; Bernardi, Francesco; V. R...., Arruda; Pinotti, Mirko | file con accesso da definire |
| 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 exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects | 2012 | E., Fernandez Alanis; Pinotti, Mirko; A., Dal Mas; Balestra, Dario; Cavallari, Nicola; Me, Rogals...ka; Bernardi, Francesco; F., Pagani | file con accesso da definire |
| AN EXON-SPECIFIC U1 SMALL NUCLEAR RNA (snRNA) STRATEGY TO CORRECT SPLICING MUTATIONS ASSOCIATED TO HEMOPHILIA B | 2012 | Balestra, Dario; Cavallari, Nicola; E., Fernandez Alanis; A., Dal Mas; M. E., Rogalska; Pagani, F...ranco; Bernardi, Francesco; Pinotti, Mirko | file con accesso da definire |
| An exon-specific U1snRNA induces a robust factor IX activity in mice expressing multiple human FIX splicing mutants | 2016 | Balestra, Dario; Scalet, Daniela; Pagani, Franco; Rogalska, Malgorzata Ewa; Mari, Rosella; Bernar...di, Francesco; 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 |
| Base and Prime Editors as new correction approaches for recurrent Hemophilia A Nonsense Mutations | 2024 | Testa, M.; Bernardi, F.; Branchini, A.; Pinotti, M.; Balestra, D. | file con accesso da definire |
| C6orf10 low-frequency and rare variants in italian multiple sclerosis patients | 2019 | Ziliotto, Nicole; Marchetti, Giovanna; Scapoli, Chiara; Bovolenta, Matteo; Meneghetti, Silvia; Be...nazzo, Andrea; Lunghi, Barbara; Balestra, Dario; Laino, Lorenza Anna; Bozzini, Nicolò; Guidi, Irene; Salvi, Fabrizio; Straudi, Sofia; Gemmati, Donato; Menegatti, Erica; Zamboni, Paolo; Bernardi, Francesco | |
| Contribution of asialoglycoprotein receptor ASGR2 5’ UTR polymorphisms to full-length FVIII concentrate pharmacokinetics | 2020 | Lunghi, B.; Morfini, M.; Martinelli, N.; Frusconi, S.; Balestra, D.; Branchini, A.; Linari, S.; M...archetti, G.; Castaman, G.; Bernardi, F. | |
| Correction of aberrant splicing causing haemophilia B through the combination of compensatory U1snRNAs and antisense oligonucleotides | 2015 | Scalet, Daniela; Balestra, Dario; Barbon, Elena; Cavallari, Nicola; Perrone, Daniela; Bernardi, F...rancesco; Pinotti, Mirko | file con accesso da definire |
| Counteracting the Common Shwachman-Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing | 2023 | Peretto, Laura; Tonetto, Elena; Maestri, Iva; Bezzerri, Valentino; Valli, Roberto; Cipolli, Marco...; Pinotti, Mirko; Balestra, Dario | |
| 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 |