An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species
Vu Hong, Ai, Suel, Laurence, Petat, Eva, Dubois, Auriane, Le Brun, Pierre-Romain, Guerchet, Nicolas, Veron, Philippe, Poupiot, Jérôme, Richard, Isabelle
Published in Nature communications (11.09.2024)
Published in Nature communications (11.09.2024)
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Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B
Ferla, Rita, Dell’Aquila, Fabio, Doria, Monica, Ferraiuolo, Maria, Noto, Alessia, Grazioli, Fabiana, Ammendola, Virginia, Testa, Francesco, Melillo, Paolo, Iodice, Carolina, Risca, Giulia, Tedesco, Novella, le Brun, Pierre Romain, Surace, Enrico Maria, Simonelli, Francesca, Galimberti, Stefania, Valsecchi, Maria Grazia, Marteau, Jean-Brice, Veron, Philippe, Colloca, Stefano, Auricchio, Alberto
Published in Molecular therapy. Methods & clinical development (09.03.2023)
Published in Molecular therapy. Methods & clinical development (09.03.2023)
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Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver
Jauze, Louisa, Vie, Mallaury, Miagoux, Quentin, Rossiaud, Lucille, Vidal, Patrice, Montalvo-Romeral, Valle, Saliba, Hanadi, Jarrige, Margot, Polveche, Helene, Nozi, Justine, Le Brun, Pierre-Romain, Bocchialini, Luca, Francois, Amandine, Cosette, Jérémie, Rouillon, Jérémy, Collaud, Fanny, Bordier, Fanny, Bertil-Froidevaux, Emilie, Georger, Christophe, van Wittenberghe, Laetitia, Miranda, Adeline, Daniele, Nathalie F, Gross, David-Alexandre, Hoch, Lucile, Nissan, Xavier, Ronzitti, Giuseppe
Published in JCI insight (16.05.2024)
Published in JCI insight (16.05.2024)
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Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI
Brunetti-Pierri, Nicola, Ferla, Rita, Ginocchio, Virginia Maria, Rossi, Alessandro, Fecarotta, Simona, Romano, Roberta, Parenti, Giancarlo, Yildiz, Yilmaz, Zancan, Stefano, Pecorella, Valentina, Dell'Anno, Margherita, Graziano, Mafalda, Alliegro, Marialuisa, Andria, Generoso, Santamaria, Francesca, Brunetti-Pierri, Raffaella, Simonelli, Francesca, Nigro, Vincenzo, Vargas, Maria, Servillo, Giuseppe, Borgia, Francesco, Soscia, Ernesto, Gargaro, Marco, Funghini, Silvia, Tedesco, Novella, Le Brun, Pierre Romain, Rupar, Charles A, Prasad, Chitra, O'Callaghan, Mar, Mitchell, John J, Danos, Olivier, Marteau, Jean-Brice, Galimberti, Stefania, Valsecchi, Maria Grazia, Veron, Philippe, Mingozzi, Federico, Fallarino, Francesca, la Marca, Giancarlo, Sivri, H Serap, Auricchio, Alberto
Published in NEJM evidence (01.07.2022)
Published in NEJM evidence (01.07.2022)
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