Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

by Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Published in Mol Ther (2016)

Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

by Polito, Vinicia Assunta, Abbondante, Serena, Polishchuk, Roman S., Nusco, Edoardo, Salvia, Rosaria, Cosma, Maria Pia
Published 2010

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)

Impaired parkin-mediated mitochondrial targeting to autophagosomes differentially contributes to tissue pathology in lysosomal storage diseases

by de Pablo-Latorre, Raquel, Saide, Assunta, Polishhuck, Elena V., Nusco, Edoardo, Fraldi, Alessandro, Ballabio, Andrea
Published 2012

Retinal Degeneration in MPS-IIIA Mouse Model

by Intartaglia, Daniela, Giamundo, Giuliana, Marrocco, Elena, Maffia, Veronica, Salierno, Francesco Giuseppe, Nusco, Edoardo, Fraldi, Alessandro, Conte, Ivan, Sorrentino, Nicolina Cristina
Published in Front Cell Dev Biol (2020)

Systemic inflammation and neurodegeneration in a mouse model of multiple sulfatase deficiency

by Settembre, Carmine, Annunziata, Ida, Spampanato, Carmine, Zarcone, Daniela, Cobellis, Gilda, Nusco, Edoardo, Zito, Ester, Tacchetti, Carlo, Cosma, Maria Pia, Ballabio, Andrea
Published 2007

Disease Rescue and Increased Lifespan in a Model of Cardiomyopathy and Muscular Dystrophy by Combined AAV Treatments

by Vitiello, Carmen, Faraso, Stefania, Sorrentino, Nicolina Cristina, Di Salvo, Giovanni, Nusco, Edoardo, Nigro, Gerardo, Cutillo, Luisa, Calabrò, Raffaele, Auricchio, Alberto, Nigro, Vincenzo
Published 2009

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

by Fraldi, Alessandro, Biffi, Alessandra, Lombardi, Alessia, Visigalli, Ilaria, Pepe, Stefano, Settembre, Carmine, Nusco, Edoardo, Auricchio, Alberto, Naldini, Luigi, Ballabio, Andrea, Cosma, Maria Pia
Published 2007

Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis

by Ferriero, Rosa, Manco, Giuseppe, Lamantea, Eleonora, Nusco, Edoardo, Ferrante, Mariella I., Sordino, Paolo, Stacpoole, Peter W., Lee, Brendan, Zeviani, Massimo, Brunetti-Pierri, Nicola
Published 2013

Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI

by Ferla, Rita, Alliegro, Marialuisa, Marteau, Jean-Brice, Dell’Anno, Margherita, Nusco, Edoardo, Pouillot, Severine, Galimberti, Stefania, Valsecchi, Maria Grazia, Zuliani, Vincent, Auricchio, Alberto
Published in Mol Ther Methods Clin Dev (2017)

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

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

Efficacy of a Combined Intracerebral and Systemic Gene Delivery Approach for the Treatment of a Severe Lysosomal Storage Disorder

by Spampanato, Carmine, De Leonibus, Elvira, Dama, Paola, Gargiulo, Annagiusi, Fraldi, Alessandro, Sorrentino, Nicolina Cristina, Russo, Fabio, Nusco, Edoardo, Auricchio, Alberto, Surace, Enrico M, Ballabio, Andrea
Published 2011

A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA

by Sorrentino, Nicolina Cristina, D'Orsi, Luca, Sambri, Irene, Nusco, Edoardo, Monaco, Ciro, Spampanato, Carmine, Polishchuk, Elena, Saccone, Paola, De Leonibus, Elvira, Ballabio, Andrea, Fraldi, Alessandro
Published 2013

Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

by Soria, Leandro R., Allegri, Gabriella, Melck, Dominique, Pastore, Nunzia, Annunziata, Patrizia, Paris, Debora, Polishchuk, Elena, Nusco, Edoardo, Thöny, Beat, Motta, Andrea, Häberle, Johannes, Ballabio, Andrea, Brunetti-Pierri, Nicola
Published in Proc Natl Acad Sci U S A (2018)

mTORC1 hyperactivation arrests bone growth in lysosomal storage disorders by suppressing autophagy

by Bartolomeo, Rosa, Cinque, Laura, De Leonibus, Chiara, Forrester, Alison, Salzano, Anna Chiara, Monfregola, Jlenia, De Gennaro, Emanuela, Nusco, Edoardo, Azario, Isabella, Lanzara, Carmela, Serafini, Marta, Levine, Beth, Ballabio, Andrea, Settembre, Carmine
Published in J Clin Invest (2017)

AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease

by Gatto, Francesca, Rossi, Barbara, Tarallo, Antonietta, Polishchuk, Elena, Polishchuk, Roman, Carrella, Alessandra, Nusco, Edoardo, Alvino, Filomena Grazia, Iacobellis, Francesca, De Leonibus, Elvira, Auricchio, Alberto, Diez-Roux, Graciana, Ballabio, Andrea, Parenti, Giancarlo
Published in Sci Rep (2017)

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease

by Monaco, Antonio, Maffia, Veronica, Sorrentino, Nicolina Cristina, Sambri, Irene, Ezhova, Yulia, Giuliano, Teresa, Cacace, Vincenzo, Nusco, Edoardo, De Risi, Maria, De Leonibus, Elvira, Schrader, Thomas, Klärner, Frank-Gerrit, Bitan, Gal, Fraldi, Alessandro
Published in Mol Ther (2020)

Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA

by Sorrentino, Nicolina Cristina, Cacace, Vincenzo, De Risi, Maria, Maffia, Veronica, Strollo, Sandra, Tedesco, Novella, Nusco, Edoardo, Romagnoli, Noemi, Ventrella, Domenico, Huang, Yan, Liu, Nan, Kalled, Susan L., Choi, Vivian W., De Leonibus, Elvira, Fraldi, Alessandro
Published in Mol Ther Methods Clin Dev (2019)

Modelling TFE renal cell carcinoma in mice reveals a critical role of WNT signaling

by Calcagnì, Alessia, kors, Lotte, Verschuren, Eric, De Cegli, Rossella, Zampelli, Nicolina, Nusco, Edoardo, Confalonieri, Stefano, Bertalot, Giovanni, Pece, Salvatore, Settembre, Carmine, Malouf, Gabriel G, Leemans, Jaklien C, de Heer, Emile, Salvatore, Marco, Peters, Dorien JM, Di Fiore, Pier Paolo, Ballabio, Andrea
Published in eLife (2016)

Light‐responsive microRNA miR‐211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearance

by Naso, Federica, Intartaglia, Daniela, Falanga, Danila, Soldati, Chiara, Polishchuk, Elena, Giamundo, Giuliana, Tiberi, Paola, Marrocco, Elena, Scudieri, Paolo, Di Malta, Chiara, Trapani, Ivana, Nusco, Edoardo, Salierno, Francesco Giuseppe, Surace, Enrico Maria, Galietta, Luis JV, Banfi, Sandro, Auricchio, Alberto, Ballabio, Andrea, Medina, Diego Luis, Conte, Ivan
Published in EMBO J (2020)