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RNA-based therapeutic approaches for blood coagulation factor deficiencies caused by a splicing mutations 2013 Balestra, Dario; Fernandez, Eugenio; Dal Mas, Andrea; Cavallari, Nicola; Barbon, Elena; Bussani, ...Erica; Ferraresi, Paolo; Canella, Alessandro; Branchini, Alessio; Campioni, Matteo; Baroni, Marcello; Mattioli, Chiara; Pagani, Franco; Pinotti, Mirko file con accesso da definire
Development of cellular and animal models of coagulation factors deficiencies for the assessment of innovative therapeutic approaches acting on transcriptional and post-transcriptional regulation 2015 Barbon, Elena
Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides 2015 Balestra, Dario; Barbon, Elena; Scalet, Daniela; Cavallari, Nicola; Perrone, Daniela; Zanibellato..., Silvia; Bernardi, Francesco; Pinotti, Mirko
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
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
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
Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics 2016 Barbon, Elena; Ferrarese, Mattia; Wittenberghe, Laetitia van; Sanatine, Peggy; Ronzitti, Giuseppe...; Collaud, Fanny; Colella, Pasqualina; Pinotti, Mirko; Mingozzi, Federico
Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein Function 2016 Tajnik, Mojca; Rogalska, Malgorzata Ewa; Bussani, Erica; Barbon, Elena; Balestra, Dario; Pinotti,... Mirko; Pagani, Franco
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System 2017 Lattanzi, Annalisa; Duguez, Stephanie; Moiani, Arianna; Izmiryan, Araksya; Barbon, Elena; Martin,... Samia; Mamchaoui, Kamel; Mouly, Vincent; Bernardi, Francesco; Mavilio, Fulvio; Bovolenta, Matteo
Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model 2018 Donadon, Irving; Pinotti, Mirko; Rajkowska, Katarzyna; Pianigiani, Giulia; Barbon, Elena; Morini,... Elisabetta; Motaln, Helena; Rogelj, Boris; Mingozzi, Federico; Slaugenhaupt, Susan A.; Pagani, Franco
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
Mostrati risultati da 1 a 12 di 12
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