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

gan Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Cyhoeddwyd yn Mol Ther (2016)

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

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

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

gan Ferriero, Rosa, Nusco, Edoardo, De Cegli, Rossella, Carissimo, Annamaria, Manco, Giuseppe, Brunetti-Pierri, Nicola
Cyhoeddwyd yn J Hepatol (2018)

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

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

Retinal Degeneration in MPS-IIIA Mouse Model

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

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

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

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

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

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

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

Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis

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

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

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

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

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

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

gan 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
Cyhoeddwyd 2011

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

gan 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
Cyhoeddwyd 2013

Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

gan 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
Cyhoeddwyd yn Proc Natl Acad Sci U S A (2018)

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

gan 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
Cyhoeddwyd yn J Clin Invest (2017)

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

gan 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
Cyhoeddwyd yn Sci Rep (2017)

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

gan 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
Cyhoeddwyd yn Mol Ther (2020)

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

gan 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
Cyhoeddwyd yn Mol Ther Methods Clin Dev (2019)

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

gan 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
Cyhoeddwyd yn eLife (2016)

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

gan 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
Cyhoeddwyd yn EMBO J (2020)