Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
Titel:
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
Auteur:
Collaud, Fanny Bortolussi, Giulia Guianvarc’h, Laurence Aronson, Sem J. Bordet, Thierry Veron, Philippe Charles, Severine Vidal, Patrice Sola, Marcelo Simon Rundwasser, Stephanie Dufour, Delphine G. Lacoste, Florence Luc, Cyril Wittenberghe, Laetitia v. Martin, Samia Le Bec, Christine Bosma, Piter J. Muro, Andres F. Ronzitti, Giuseppe Hebben, Matthias Mingozzi, Federico