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Pasquale Piccolo
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Wilson disease protein ATP7B utilizes lysosomal exocytosis to maintain copper homeostasis
EV Polishchuk, M Concilli, S Iacobacci, G Chesi, N Pastore, P Piccolo, ...
Developmental cell 29 (6), 686-700, 2014
2602014
Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha‐1‐anti‐trypsin deficiency
N Pastore, K Blomenkamp, F Annunziata, P Piccolo, P Mithbaokar, ...
EMBO molecular medicine 5 (3), 397-412, 2013
1542013
Supravalvular aortic stenosis: elastin arteriopathy
G Merla, N Brunetti-Pierri, P Piccolo, L Micale, MN Loviglio
Circulation: Cardiovascular Genetics 5 (6), 692-696, 2012
1222012
Retinal transduction profiles by high-capacity viral vectors
A Puppo, G Cesi, E Marrocco, P Piccolo, S Jacca, DM Shayakhmetov, ...
Gene therapy 21 (10), 855-865, 2014
652014
SR-A and SREC-I are Kupffer and endothelial cell receptors for helper-dependent adenoviral vectors
P Piccolo, F Vetrini, P Mithbaokar, NC Grove, T Bertin, D Palmer, P Ng, ...
Molecular Therapy 21 (4), 767-774, 2013
592013
Slc7a7 disruption causes fetal growth retardation by downregulating Igf1 in the mouse model of lysinuric protein intolerance
MP Sperandeo, P Annunziata, A Bozzato, P Piccolo, L Maiuri, ...
American Journal of Physiology-Cell Physiology 293 (1), C191-C198, 2007
512007
Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1
R Castello, R Borzone, S D'Aria, P Annunziata, P Piccolo, N Brunetti-Pierri
Gene therapy 23 (2), 129-134, 2016
482016
Balloon catheter delivery of helper-dependent adenoviral vector results in sustained, therapeutic hFIX expression in rhesus macaques
N Brunetti-Pierri, A Liou, P Patel, D Palmer, N Grove, M Finegold, ...
Molecular therapy 20 (10), 1863-1870, 2012
452012
SMAD4 mutations causing Myhre syndrome result in disorganization of extracellular matrix improved by losartan
P Piccolo, P Mithbaokar, V Sabatino, J Tolmie, D Melis, MC Schiaffino, ...
European Journal of Human Genetics 22 (8), 988-994, 2014
362014
Down‐regulation of hepatocyte nuclear factor‐4α and defective zonation in livers expressing mutant Z α1‐antitrypsin
P Piccolo, P Annunziata, LR Soria, S Attanasio, A Barbato, R Castello, ...
Hepatology 66 (1), 124-135, 2017
292017
SR-A and SREC-I binding peptides increase HDAd-mediated liver transduction
P Piccolo, P Annunziata, P Mithbaokar, N Brunetti-Pierri
Gene therapy 21 (11), 950-957, 2014
222014
Up-regulation of miR-34b/c by JNK and FOXO3 protects from liver fibrosis
P Piccolo, R Ferriero, A Barbato, S Attanasio, M Monti, C Perna, F Borel, ...
Proceedings of the National Academy of Sciences 118 (10), e2025242118, 2021
202021
Gene therapy for inherited diseases of liver metabolism
P Piccolo, N Brunetti-Pierri
Human gene therapy 26 (4), 186-192, 2015
202015
The growth hormone-insulin-like growth factor axis in glycogen storage disease type 1: evidence of different growth patterns and insulin-like growth factor levels in patients …
D Melis, R Pivonello, G Parenti, R Della Casa, M Salerno, F Balivo, ...
The Journal of pediatrics 156 (4), 663-670. e1, 2010
202010
CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicity
S Attanasio, R Ferriero, G Gernoux, R De Cegli, A Carissimo, E Nusco, ...
Journal of Biological Chemistry 295 (38), 13213-13223, 2020
192020
MIB2 variants altering NOTCH signalling result in left ventricle hypertrabeculation/non-compaction and are associated with Ménétrier-like gastropathy
P Piccolo, S Attanasio, I Secco, R Sangermano, C Strisciuglio, ...
Human Molecular Genetics 26 (1), 33-43, 2017
152017
Geleophysic dysplasia: novel missense variants and insights into ADAMTSL2 intracellular trafficking
P Piccolo, V Sabatino, P Mithbaokar, E Polishchuck, SK Law, ...
Molecular genetics and metabolism reports 21, 100504, 2019
142019
Intrathecal injection of helper-dependent adenoviral vectors results in long-term transgene expression in neuroependymal cells and neurons
S Dindot, P Piccolo, N Grove, D Palmer, N Brunetti-Pierri
Human gene therapy 22 (6), 745-751, 2011
142011
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice
A Padula, R Petruzzelli, SA Philbert, SJ Church, F Esposito, S Campione, ...
Molecular Therapy-Methods & Clinical Development 26, 495-504, 2022
132022
Challenges and prospects for helper-dependent adenoviral vector-mediated gene therapy
P Piccolo, N Brunetti-Pierri
Biomedicines 2 (2), 132-148, 2014
132014
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