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

Yazar: Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Yayımlandı Mol Ther (2016)

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

Yazar: Polito, Vinicia Assunta, Abbondante, Serena, Polishchuk, Roman S., Nusco, Edoardo, Salvia, Rosaria, Cosma, Maria Pia
Baskı/Yayın Bilgisi 2010

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

Yazar: Ferriero, Rosa, Nusco, Edoardo, De Cegli, Rossella, Carissimo, Annamaria, Manco, Giuseppe, Brunetti-Pierri, Nicola
Yayımlandı J Hepatol (2018)

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

Yazar: de Pablo-Latorre, Raquel, Saide, Assunta, Polishhuck, Elena V., Nusco, Edoardo, Fraldi, Alessandro, Ballabio, Andrea
Baskı/Yayın Bilgisi 2012

Retinal Degeneration in MPS-IIIA Mouse Model

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

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

Yazar: Settembre, Carmine, Annunziata, Ida, Spampanato, Carmine, Zarcone, Daniela, Cobellis, Gilda, Nusco, Edoardo, Zito, Ester, Tacchetti, Carlo, Cosma, Maria Pia, Ballabio, Andrea
Baskı/Yayın Bilgisi 2007

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

Yazar: Vitiello, Carmen, Faraso, Stefania, Sorrentino, Nicolina Cristina, Di Salvo, Giovanni, Nusco, Edoardo, Nigro, Gerardo, Cutillo, Luisa, Calabrò, Raffaele, Auricchio, Alberto, Nigro, Vincenzo
Baskı/Yayın Bilgisi 2009

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

Yazar: Fraldi, Alessandro, Biffi, Alessandra, Lombardi, Alessia, Visigalli, Ilaria, Pepe, Stefano, Settembre, Carmine, Nusco, Edoardo, Auricchio, Alberto, Naldini, Luigi, Ballabio, Andrea, Cosma, Maria Pia
Baskı/Yayın Bilgisi 2007

Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis

Yazar: Ferriero, Rosa, Manco, Giuseppe, Lamantea, Eleonora, Nusco, Edoardo, Ferrante, Mariella I., Sordino, Paolo, Stacpoole, Peter W., Lee, Brendan, Zeviani, Massimo, Brunetti-Pierri, Nicola
Baskı/Yayın Bilgisi 2013

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

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

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

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

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

Yazar: 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
Baskı/Yayın Bilgisi 2011

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

Yazar: 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
Baskı/Yayın Bilgisi 2013

Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

Yazar: 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
Yayımlandı Proc Natl Acad Sci U S A (2018)

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

Yazar: 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
Yayımlandı J Clin Invest (2017)

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

Yazar: 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
Yayımlandı Sci Rep (2017)

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

Yazar: 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
Yayımlandı Mol Ther (2020)

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

Yazar: 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
Yayımlandı Mol Ther Methods Clin Dev (2019)

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

Yazar: 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
Yayımlandı eLife (2016)

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

Yazar: 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
Yayımlandı EMBO J (2020)