Challenges and Prospects for Helper-Dependent Adenoviral Vector-Mediated Gene Therapy

av Piccolo, Pasquale, Brunetti-Pierri, Nicola
I publikationen Biomedicines (2014)

SR-A and SREC-I binding peptides increase HDAd-mediated liver transduction

av Piccolo, Pasquale, Annunziata, Patrizia, Mithbaokar, Pratibha, Brunetti-Pierri, Nicola
I publikationen Gene Ther (2014)

Intrathecal Injection of Helper-Dependent Adenoviral Vectors Results in Long-Term Transgene Expression in Neuroependymal Cells and Neurons

av Dindot, Scott, Piccolo, Pasquale, Grove, Nathan, Palmer, Donna, Brunetti-Pierri, Nicola
Publicerad 2011

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

av Castello, Raffaele, Borzone, Roberta, D’Aria, Stefania, Annunziata, Patrizia, Piccolo, Pasquale, Brunetti-Pierri, Nicola
I publikationen Gene Ther (2015)

SR-A and SREC-I Are Kupffer and Endothelial Cell Receptors for Helper-dependent Adenoviral Vectors

av Piccolo, Pasquale, Vetrini, Francesco, Mithbaokar, Pratibha, Grove, Nathan C, Bertin, Terry, Palmer, Donna, Ng, Philip, Brunetti-Pierri, Nicola
Publicerad 2013

Skin fibroblasts of patients with geleophysic dysplasia due to FBN1 mutations have lysosomal inclusions and losartan improves their microfibril deposition defect

av Piccolo, Pasquale, Sabatino, Valeria, Mithbaokar, Pratibha, Polishchuk, Elena, Hicks, John, Polishchuk, Roman, Bacino, Carlos A., Brunetti‐Pierri, Nicola
I publikationen Mol Genet Genomic Med (2019)

Terminal Osseous Dysplasia With Pigmentary Defects (TODPD): Follow-Up of the First Reported Family, Characterization of the Radiological Phenotype, and Refinement of the Linkage Re...

av Brunetti-Pierri, Nicola, Lachman, Ralph, Lee, Kwanghyuk, Leal, Suzanne M., Piccolo, Pasquale, Van den Veyver, Ignatia B., Bacino, Carlos A.
Publicerad 2010

SMAD4 mutations causing Myhre syndrome result in disorganization of extracellular matrix improved by losartan

av Piccolo, Pasquale, Mithbaokar, Pratibha, Sabatino, Valeria, Tolmie, John, Melis, Daniela, Schiaffino, Maria Cristina, Filocamo, Mirella, Andria, Generoso, Brunetti-Pierri, Nicola
Publicerad 2014

Geleophysic dysplasia: novel missense variants and insights into ADAMTSL2 intracellular trafficking

av Piccolo, Pasquale, Sabatino, Valeria, Mithbaokar, Pratibha, Polishchuck, Elena, Law, Simon K., Magraner-Pardo, Lorena, Pons, Tirso, Polishchuck, Roman, Brunetti-Pierri, Nicola
I publikationen Mol Genet Metab Rep (2019)

Terminal osseous dysplasia with pigmentary defects (TODPD) due to a recurrent filamin A (FLNA) mutation

av Brunetti-Pierri, Nicola, Torrado, Maria, Fernandez, Maria del Carmen, Tello, Ana Maria, Arberas, Claudia L, Cardinale, Antonella, Piccolo, Pasquale, Bacino, Carlos A
I publikationen Mol Genet Genomic Med (2014)

Cutis laxa and fatal pulmonary hypertension: a newly recognized syndrome?

av Brunetti-Pierri, Nicola, Piccolo, Pasquale, Morava, Eva, Wevers, Ron A., McGuirk, Megan, Johnson, Yvette R., Urban, Zsolt, Dishop, Megan K., Potocki, Lorraine
I publikationen Clin Dysmorphol (2011)

CHOP and c-JUN up-regulate the mutant Z α(1)-antitrypsin, exacerbating its aggregation and liver proteotoxicity

av Attanasio, Sergio, Ferriero, Rosa, Gernoux, Gwladys, De Cegli, Rossella, Carissimo, Annamaria, Nusco, Edoardo, Campione, Severo, Teckman, Jeffrey, Mueller, Christian, Piccolo, Pasquale, Brunetti-Pierri, Nicola
I publikationen J Biol Chem (2020)

Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques

av Brunetti-Pierri, Nicola, Liou, Aimee, Patel, Priti, Palmer, Donna, Grove, Nathan, Finegold, Milton, Piccolo, Pasquale, Donnachie, Elizabeth, Rice, Karen, Beaudet, Arthur, Mullins, Charles, Ng, Philip
Publicerad 2012

Retinal transduction profiles by high-capacity viral vectors

av Puppo, Agostina, Cesi, Giulia, Marrocco, Elena, Piccolo, Pasquale, Jacca, Sarah, Shayakhmetov, Dmitry M., Parks, Robin J., Davidson, Beverly L., Colloca, Stefano, Brunetti-Pierri, Nicola, Ng, Philip, Donofrio, Gaetano, Auricchio, Alberto
Publicerad 2014

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency

av Pastore, Nunzia, Blomenkamp, Keith, Annunziata, Fabio, Piccolo, Pasquale, Mithbaokar, Pratibha, Maria Sepe, Rosa, Vetrini, Francesco, Palmer, Donna, Ng, Philip, Polishchuk, Elena, Iacobacci, Simona, Polishchuk, Roman, Teckman, Jeffrey, Ballabio, Andrea, Brunetti-Pierri, Nicola
Publicerad 2013

Up-regulation of miR-34b/c by JNK and FOXO3 protects from liver fibrosis

av Piccolo, Pasquale, Ferriero, Rosa, Barbato, Anna, Attanasio, Sergio, Monti, Marcello, Perna, Claudia, Borel, Florie, Annunziata, Patrizia, Carissimo, Annamaria, De Cegli, Rossella, Quagliata, Luca, Terracciano, Luigi M., Housset, Chantal, Teckman, Jeffrey H., Mueller, Christian, Brunetti-Pierri, Nicola
I publikationen Proc Natl Acad Sci U S A (2021)

Wilson Disease Protein ATP7B Utilizes Lysosomal Exocytosis to Maintain Copper Homeostasis

av Polishchuk, Elena V., Concilli, Mafalda, Iacobacci, Simona, Chesi, Giancarlo, Pastore, Nunzia, Piccolo, Pasquale, Paladino, Simona, Baldantoni, Daniela, van IJzendoorn, Sven C.D., Chan, Jefferson, Chang, Christopher J., Amoresano, Angela, Pane, Francesca, Pucci, Piero, Tarallo, Antonietta, Parenti, Giancarlo, Brunetti-Pierri, Nicola, Settembre, Carmine, Ballabio, Andrea, Polishchuk, Roman S.
Publicerad 2014