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

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

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

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

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

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

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

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

Retinal Degeneration in MPS-IIIA Mouse Model

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

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

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

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

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

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

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

Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis

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

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

di Ferla, Rita, Alliegro, Marialuisa, Marteau, Jean-Brice, Dell’Anno, Margherita, Nusco, Edoardo, Pouillot, Severine, Galimberti, Stefania, Valsecchi, Maria Grazia, Zuliani, Vincent, Auricchio, Alberto
Pubblicato 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

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

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

di 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
Pubblicazione 2011

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

di 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
Pubblicazione 2013

Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

di 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
Pubblicato in Proc Natl Acad Sci U S A (2018)

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

di 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
Pubblicato 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

di 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
Pubblicato in Sci Rep (2017)

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

di 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
Pubblicato in Mol Ther (2020)

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

di 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
Pubblicato in Mol Ther Methods Clin Dev (2019)

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

di 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
Pubblicato in eLife (2016)

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

di 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
Pubblicato in EMBO J (2020)