Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1

por Castello, Raffaele, Borzone, Roberta, D’Aria, Stefania, Annunziata, Patrizia, Piccolo, Pasquale, Brunetti-Pierri, Nicola
Publicado 2015

Activation of the c‐Jun N‐terminal kinase pathway aggravates proteotoxicity of hepatic mutant Z alpha1‐antitrypsin

por Pastore, Nunzia, Attanasio, Sergio, Granese, Barbara, Castello, Raffaele, Teckman, Jeffrey, Wilson, Andrew A., Ballabio, Andrea, Brunetti‐Pierri, Nicola
Publicado 2017

In Silico Modeling of Liver Metabolism in a Human Disease Reveals a Key Enzyme for Histidine and Histamine Homeostasis

por Pagliarini, Roberto, Castello, Raffaele, Napolitano, Francesco, Borzone, Roberta, Annunziata, Patrizia, Mandrile, Giorgia, De Marchi, Mario, Brunetti-Pierri, Nicola, di Bernardo, Diego
Publicado 2016

Retinal dystrophy in an individual carrying a de novo missense variant of SMARCA4

por Cappuccio, Gerarda, Brunetti‐Pierri, Raffaella, Torella, Annalaura, Pinelli, Michele, Castello, Raffaele, Casari, Giorgio, Nigro, Vincenzo, Banfi, Sandro, Simonelli, Francesca, Brunetti‐Pierri, Nicola
Publicado 2019