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Mostrati risultati da 21 a 40 di 67
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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
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
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
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
Mcl-1 involvement in mitochondrial dynamics is associated with apoptotic cell death 2016 Morciano, Giampaolo; Giorgi, Carlotta; Balestra, Dario; Marchi, Saverio; Perrone, Daniela; Pinott...i, Mirko; Pinton, Paolo
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
MOLECULAR MECHANISMS AND THERAPEUTIC APROACHES FOR RESTORATION OF mRNA TRANSCRIPTION, MATURATION AND TRANSLATION IN INHERITED COAGULATION FACTOR DEFICIENCIES 2016 Balestra, Dario; Bovolenta, Matteo; Branchini, Alessio; Pinotti, Mirko; Bernardi, Francesco file con accesso da definire
F8 splicing-swithcing molecules for tailored hemophilia A therapies 2017 Balestra, Dario file con accesso da definire
Exploring Splicing-Switching Molecules For Seckel Syndrome Therapy 2017 Scalet, Daniela; Balestra, Dario; Rohban, Sara; Bovolenta, Matteo; Perrone, Daniela; Bernardi, Fr...ancesco; Campaner, Stefano; Pinotti, Mirko
Secretion of wild-type factor IX upon readthrough over F9 pre-peptide nonsense mutations causing hemophilia B 2018 Ferrarese, Mattia; Testa, Maria Francesca; Balestra, Dario; Bernardi, Francesco; Pinotti, Mirko; ...Branchini, Alessio
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
Rescue of missense and splicing mutations in Haemophilia A by a unique Exon Specific U1snRNA 2018 Balestra, Dario; Scalet, Daniela; Leo, Gabriele; Donadon, I.; Lombardi, Silvia; Bernardi, Frances...co; Pinotti, M.
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
La terapia genica nelle Malattie emorragiche e trombotiche 2018 Bernardi, Francesco; Balestra, Dario file con accesso da definire
Elucidation of aberrant SBDS splicing mechanisms to design RNA-therapeutics for Shwachman-Diamond Syndrome 2018 Balestra, Dario file con accesso da definire
RNA-Based therapeutics for OTC deficiency 2018 Balestra, Dario file con accesso da definire
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
Secretion of wild-type factor IX upon readthrough over F9 pre-peptide nonsense mutations causing hemophilia B 2018 Ferrarese, Mattia; Testa, Maria Francesca; Balestra, Dario; Bernardi, Francesco; Pinotti, Mirko; ...Branchini, Alessio
The somatic FAH C.1061C>A change counteracts the frequent FAH c.1062+5G>A mutation and permits U1snRNA-based splicing correction 2018 Scalet, Daniela; Sacchetto, Claudia; Bernardi, Francesco; Pinotti, Mirko; Van De Graaf, Stan F. J....; Balestra, Dario
Mostrati risultati da 21 a 40 di 67
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