Adeno-associated virus vector mediated gene therapy for Crigler Najjar syndrome: preliminary report of safety and efficacy from the CareCN clinical trial

D'Antiga;L (D'Antiga;Lorenzo) Beuers;U (Beuers;Ulrich) Brunetti-Pierri;N (Brunetti-Pierri;Nicola) Baumann;U (Baumann;Ulrich);Di Giorgio, Angelo;Aronson;SJ (Aronson;Sem J. );Hubert;A (Hubert;Aurelie) Romano;R (Romano;Roberta) Junge;N (Junge;Norman) Bosma;P (Bosma;Piter) Pavlyuk;M (Pavlyuk;Mariya) Veron;P (Veron;Philippe) Ronzitti;G (Ronzitti;Giuseppe) Collaud;F (Collaud;Fanny) Honnet;G (Honnet;Geraldine) Knuchel-Legendre;N (Knuchel-Legendre;Nathalie) Mingozzi;F (Mingozzi;Federico) Labrune;P (Labrune;Philippe)

2021-01-01