Sfoglia per Autore
Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease
2023 Padula, Agnese; Spinelli, Michele; Nusco, Edoardo; Bujanda Cundin, Xabier; Capolongo, Filomena; Campione, Severo; Perna, Claudia; Bastille, Amy; Ericson, Megan E; Wang, Chih-Chieh; Zhang, Shengwen; Amoresano, Angela; Nacht, Mariana; Piccolo, Pasquale
CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicity
2020 Attanasio, S.; Ferriero, R.; Gernoux, G.; De Cegli, R.; Carissimo, A.; Nusco, E.; Campione, S.; Teckman, J.; Mueller, C.; Piccolo, P.; Brunetti-Pierri, Nicola
Skin fibroblasts of patients with geleophysic dysplasia due to FBN1 mutations have lysosomal inclusions and losartan improves their microfibril deposition defect
2019 Piccolo, Pasquale; Sabatino, Valeria; Mithbaokar, Pratibha; Polishchuk, Elena; Hicks, John; Polishchuk, Roman; Bacino, Carlos A; Brunetti-Pierri, Nicola
MIB2 variants altering NOTCH signalling result in left ventricle hypertrabeculation/non-compaction and are associated with Ménétrier-like gastropathy
2017 Piccolo, Pasquale; Attanasio, Sergio; Secco, Ilaria; Sangermano, Riccardo; Strisciuglio, Caterina; Limongelli, Giuseppe; Miele, Erasmo; Mutarelli, Margherita; Banfi, Sandro; Nigro, Vincenzo; Pons, Tirso; Valencia, Alfonso; Zentilin, Lorena; Campione, Severo; Nardone, Gerardo; Lynnes, Ty C; Celestino-Soper, Patricia B S; Spoonamore, Katherine G; D'Armiento, Francesco P; Giacca, Mauro; Staiano, Annamaria; Vatta, Matteo; Collesi, Chiara; Brunetti-Pierri, Nicola
Gene therapy for inherited diseases of liver metabolism.
2015 Piccolo, P; BRUNETTI PIERRI, Nicola
Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1
2015 Castello, R; Borzone, Roberta; D'Aria, S; Annunziata, P; Piccolo, P; BRUNETTI PIERRI, Nicola
| Titolo | Tipologia | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|---|
| Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease | 1.1 Articolo in rivista | 2023 | Padula, Agnese; Spinelli, Michele; Nusco, Edoardo; Bujanda Cundin, Xabier; Capolongo, Filomena; Campione, Severo; Perna, Claudia; Bastille, Amy; Ericson, Megan E; Wang, Chih-Chieh; Zhang, Shengwen; Amoresano, Angela; Nacht, Mariana; Piccolo, Pasquale | |
| CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicity | 1.1 Articolo in rivista | 2020 | Attanasio, S.; Ferriero, R.; Gernoux, G.; De Cegli, R.; Carissimo, A.; Nusco, E.; Campione, S.; Teckman, J.; Mueller, C.; Piccolo, P.; Brunetti-Pierri, Nicola | |
| Skin fibroblasts of patients with geleophysic dysplasia due to FBN1 mutations have lysosomal inclusions and losartan improves their microfibril deposition defect | 1.1 Articolo in rivista | 2019 | Piccolo, Pasquale; Sabatino, Valeria; Mithbaokar, Pratibha; Polishchuk, Elena; Hicks, John; Polishchuk, Roman; Bacino, Carlos A; Brunetti-Pierri, Nicola | |
| MIB2 variants altering NOTCH signalling result in left ventricle hypertrabeculation/non-compaction and are associated with Ménétrier-like gastropathy | 1.1 Articolo in rivista | 2017 | Piccolo, Pasquale; Attanasio, Sergio; Secco, Ilaria; Sangermano, Riccardo; Strisciuglio, Caterina; Limongelli, Giuseppe; Miele, Erasmo; Mutarelli, Margherita; Banfi, Sandro; Nigro, Vincenzo; Pons, Tirso; Valencia, Alfonso; Zentilin, Lorena; Campione, Severo; Nardone, Gerardo; Lynnes, Ty C; Celestino-Soper, Patricia B S; Spoonamore, Katherine G; D'Armiento, Francesco P; Giacca, Mauro; Staiano, Annamaria; Vatta, Matteo; Collesi, Chiara; Brunetti-Pierri, Nicola | |
| Gene therapy for inherited diseases of liver metabolism. | 1.1 Articolo in rivista | 2015 | Piccolo, P; BRUNETTI PIERRI, Nicola | |
| Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1 | 1.1 Articolo in rivista | 2015 | Castello, R; Borzone, Roberta; D'Aria, S; Annunziata, P; Piccolo, P; BRUNETTI PIERRI, Nicola |
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