AAV-Directed Liver Gene Therapy for Crigler-Najjar Syndrome

Collaud, F; Ronzitti, G; Bortolussi, G; Guianvarc'h, L; Veron, P; Charles, S; Vidal, P; Sola, MS; Leborgne, C; Rundwasser, S; Dejoint, L; Goncalves, D; Van Wittenberghe, L; Martin, S; Le Bec, C; Bosma, PJ; Muro, AF; Hebben, M; Mingozzi, F

MOLECULAR THERAPY, 2018; 26 (5): 385