BALESTRA, Dario

BALESTRA, Dario  

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 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
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
Delivery of a modified U1 small nuclear RNA alleviates splicing-defective coagulation factor VII expression in mouse models 2012 Balestra, Dario; A., Faella; N., Cavallari; P., Margaritis; F., Pagani; Bernardi, Francesco; V. R...., Arruda; Pinotti, Mirko file con accesso da definire