PICCOLO, PASQUALE
PICCOLO, PASQUALE
DIPARTIMENTO DI SCIENZE MEDICHE TRASLAZIONALI
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
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
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
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases
2024 Esposito, Federica; Dell'Aquila, Fabio; Rhiel, Manuel; Auricchio, Stefano; Chmielewski, Kay Ole; Andrieux, Geoffroy; Ferla, Rita; Horrach, Paula Sureda; Padmanabhan, Arjun; Di Cunto, Roberto; Notaro, Simone; Santeularia, Manel Llado; Boerries, Melanie; Dell'Anno, Margherita; Nusco, Edoardo; Padula, Agnese; Nutarelli, Sofia; Cornu, Tatjana I; Sorrentino, Nicolina Cristina; Piccolo, Pasquale; Trapani, Ivana; Cathomen, Toni; Auricchio, Alberto
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA
2024 Giaccio, Marianna; Monaco, Antonio; Galiano, Laura; Parente, Andrea; Borzacchiello, Luigi; Rubino, Riccardo; Klärner, Frank-Gerrit; Killa, Dennis; Perna, Claudia; Piccolo, Pasquale; Marotta, Marcello; Pan, Xuefang; Khijniak, Marie; Siddique, Ibrar; Schrader, Thomas; Alexey V., Pshezhetsky; Sorrentino, NICOLINA CRISTINA; Bitan, Gal; Fraldi, Alessandro
| Titolo | Tipologia | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|---|
| 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 | |
| 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 | |
| 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 | |
| Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases | 1.1 Articolo in rivista | 2024 | Esposito, Federica; Dell'Aquila, Fabio; Rhiel, Manuel; Auricchio, Stefano; Chmielewski, Kay Ole; Andrieux, Geoffroy; Ferla, Rita; Horrach, Paula Sureda; Padmanabhan, Arjun; Di Cunto, Roberto; Notaro, Simone; Santeularia, Manel Llado; Boerries, Melanie; Dell'Anno, Margherita; Nusco, Edoardo; Padula, Agnese; Nutarelli, Sofia; Cornu, Tatjana I; Sorrentino, Nicolina Cristina; Piccolo, Pasquale; Trapani, Ivana; Cathomen, Toni; Auricchio, Alberto | |
| Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA | 1.1 Articolo in rivista | 2024 | Giaccio, Marianna; Monaco, Antonio; Galiano, Laura; Parente, Andrea; Borzacchiello, Luigi; Rubino, Riccardo; Klärner, Frank-Gerrit; Killa, Dennis; Perna, Claudia; Piccolo, Pasquale; Marotta, Marcello; Pan, Xuefang; Khijniak, Marie; Siddique, Ibrar; Schrader, Thomas; Alexey V., Pshezhetsky; Sorrentino, NICOLINA CRISTINA; Bitan, Gal; Fraldi, Alessandro |