Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Pierre Romain le Brun"'
Autor:
Rita Ferla, Fabio Dell’Aquila, Monica Doria, Maria Ferraiuolo, Alessia Noto, Fabiana Grazioli, Virginia Ammendola, Francesco Testa, Paolo Melillo, Carolina Iodice, Giulia Risca, Novella Tedesco, Pierre Romain le Brun, Enrico Maria Surace, Francesca Simonelli, Stefania Galimberti, Maria Grazia Valsecchi, Jean-Brice Marteau, Philippe Veron, Stefano Colloca, Alberto Auricchio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 396-411 (2023)
Gene therapy of Usher syndrome type 1B (USH1B) due to mutations in the large Myosin VIIA (MYO7A) gene is limited by the packaging capacity of adeno-associated viral (AAV) vectors. To overcome this, we have previously developed dual AAV8 vectors which
Externí odkaz:
https://doaj.org/article/61581d1038c94d21b99808688a92c247
Autor:
Ai Vu Hong, Laurence Suel, Eva Petat, Auriane Dubois, Pierre-Romain Le Brun, Nicolas Guerchet, Philippe Veron, Jérôme Poupiot, Isabelle Richard
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-20 (2024)
Abstract Current adeno-associated virus (AAV) gene therapy using nature-derived AAVs is limited by non-optimal tissue targeting. In the treatment of muscular diseases (MD), high doses are often required but can lead to severe adverse effects. Here, w
Externí odkaz:
https://doaj.org/article/2e8bc0e4254344a1a716b976440ae501
Autor:
Nicola Brunetti-Pierri, Rita Ferla, Virginia Maria Ginocchio, Alessandro Rossi, Simona Fecarotta, Roberta Romano, Giancarlo Parenti, Yilmaz Yildiz, Stefano Zancan, Valentina Pecorella, Margherita Dell’Anno, Mafalda Graziano, Marialuisa Alliegro, Generoso Andria, Francesca Santamaria, Raffaella Brunetti-Pierri, Francesca Simonelli, Vincenzo Nigro, Maria Vargas, Giuseppe Servillo, Francesco Borgia, Ernesto Soscia, Marco Gargaro, Silvia Funghini, Novella Tedesco, Pierre Romain Le Brun, Charles A. Rupar, Chitra Prasad, Mar O’Callaghan, John J. Mitchell, Olivier Danos, Jean-Brice Marteau, Stefania Galimberti, Maria Grazia Valsecchi, Philippe Veron, Federico Mingozzi, Francesca Fallarino, Giancarlo la Marca, H. Serap Sivri, Alberto Auricchio
Publikováno v:
NEJM Evidence. 1
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::93d416c60dd7074db1fccb900afbb5ac
https://doi.org/10.1056/evidoa2200052
https://doi.org/10.1056/evidoa2200052