Gene therapy for inborn errors of liver metabolism: progress towards clinical applications

by Brunetti-Pierri, Nicola
Published 2008

Helper-dependent adenoviral vectors for liver-directed gene therapy

by Brunetti-Pierri, Nicola, Ng, Philip
Published 2011

Phenylbutyrate increases activity of pyruvate dehydrogenase complex

by Ferriero, Rosa, Brunetti-Pierri, Nicola
Published 2013

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

by Piccolo, Pasquale, Brunetti-Pierri, Nicola
Published in Biomedicines (2014)

Targeting autophagy for therapy of hyperammonemia

by Soria, Leandro R., Brunetti-Pierri, Nicola
Published in Autophagy (2018)

Autophagy master regulator TFEB induces clearance of toxic SERPINA1/α-1-antitrypsin polymers

by Pastore, Nunzia, Ballabio, Andrea, Brunetti-Pierri, Nicola
Published 2013

Reply to Amor et al

by Brunetti-Pierri, Nicola, Cheung, Sau Wai, Stankiewicz, Paweł
Published 2012

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

by Piccolo, Pasquale, Annunziata, Patrizia, Mithbaokar, Pratibha, Brunetti-Pierri, Nicola
Published in Gene Ther (2014)

Differential inhibition of PDKs by phenylbutyrate and enhancement of pyruvate dehydrogenase complex activity by combination with dichloroacetate

by Ferriero, Rosa, Iannuzzi, Clara, Manco, Giuseppe, Brunetti-Pierri, Nicola
Published in J Inherit Metab Dis (2015)

Systemic hypertension in two patients with ASL deficiency: A result of nitric oxide deficiency?

by Brunetti-Pierri, Nicola, Erez, Ayelet, Shchelochkov, Oleg, Craigen, William, Lee, Brendan
Published 2009

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

by Dindot, Scott, Piccolo, Pasquale, Grove, Nathan, Palmer, Donna, Brunetti-Pierri, Nicola
Published 2011

Severe presentation and complex brain malformations in an individual carrying a CCND2 variant

by Cappuccio, Gerarda, Ugga, Lorenzo, Parrini, Elena, D’Amico, Alessandra, Brunetti‐Pierri, Nicola
Published in Mol Genet Genomic Med (2019)

Correction of Hyperbilirubinemia in Gunn Rats Using Clinically Relevant Low Doses of Helper-Dependent Adenoviral Vectors

by Dimmock, David, Brunetti-Pierri, Nicola, Palmer, Donna J., Beaudet, Arthur L., Ng, Philip
Published 2011

Low-Dose Amitriptyline-Induced Acute Dystonia in a Patient with Metachromatic Leukodystrophy

by Cappuccio, Gerarda, Brunetti-Pierri, Nicola, Terrone, Gaetano, Romano, Alfonso, Andria, Generoso, Del Giudice, Ennio
Published 2012

WDR35 Mutation in Siblings with Sensenbrenner Syndrome: A Ciliopathy With Variable Phenotype

by Bacino, Carlos A., Dhar, Shweta U., Brunetti-Pierri, Nicola, Lee, Brendan, Bonnen, Penelope E.
Published 2012

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

by Castello, Raffaele, Borzone, Roberta, D’Aria, Stefania, Annunziata, Patrizia, Piccolo, Pasquale, Brunetti-Pierri, Nicola
Published in Gene Ther (2015)

Pyruvate dehydrogenase complex and lactate dehydrogenase are targets for therapy of acute liver failure

by Ferriero, Rosa, Nusco, Edoardo, De Cegli, Rossella, Carissimo, Annamaria, Manco, Giuseppe, Brunetti-Pierri, Nicola
Published in J Hepatol (2018)

Sphingolipid Metabolism Perturbations in Rett Syndrome

by Cappuccio, Gerarda, Donti, Taraka, Pinelli, Michele, Bernardo, Pia, Bravaccio, Carmela, Elsea, Sarah H., Brunetti-Pierri, Nicola
Published in Metabolites (2019)

Spondylocarpotarsal synostosis: long-term follow-up of a case due to FLNB mutations

by Brunetti-Pierri, Nicola, Esposito, Valentina, De Brasi, Daniele, Mattiacci, Dario Maria, Krakow, Deborah, Lee, Brendan, Salerno, Mariacarolina
Published 2008

Infiltrating giant cell tumor in a case of Paget’s disease of bone

by Nuzzo, Vincenzo, Ferrara, T., Zuccoli, Alfonso, Brunetti-Pierri, Raffaella, De Rosa, Roberto, Falchetti, Alberto, Franco, R., Brunetti-Pierri, Nicola
Published 2009