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

Autor Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Vydáno v Mol Ther (2016)

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

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

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

Autor Ferriero, Rosa, Nusco, Edoardo, De Cegli, Rossella, Carissimo, Annamaria, Manco, Giuseppe, Brunetti-Pierri, Nicola
Vydáno v J Hepatol (2018)

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

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

Retinal Degeneration in MPS-IIIA Mouse Model

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

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

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

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

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

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

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

Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis

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

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

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

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

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

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

Autor 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
Vydáno 2011

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

Autor 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
Vydáno 2013

Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

Autor 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
Vydáno v Proc Natl Acad Sci U S A (2018)

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

Autor 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
Vydáno v J Clin Invest (2017)

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

Autor 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
Vydáno v Sci Rep (2017)

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

Autor 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
Vydáno v Mol Ther (2020)

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

Autor 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
Vydáno v Mol Ther Methods Clin Dev (2019)

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

Autor 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
Vydáno v eLife (2016)

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

Autor 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
Vydáno v EMBO J (2020)