Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal - Banchi, Elena Gaia; Alonso, Rafael; Jacquot, Sylvie; Fransquin, Kevin; Courtot, Enora; de Saint Denis, Timothee; Ausseil, Jérôme; Piguet, Françoise (Molecular genetics and metabolism, 2024)