Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in large animal

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Bibliographic Details
Published in:Molecular genetics and metabolism 2024-02, Vol.141 (2), p.107768, Article 107768
Main Authors: Banchi, Elena Gaia, Alonso, Rafael, Jacquot, Sylvie, Fransquin, Kevin, Courtot, Enora, de Saint Denis, Timothee, Ausseil, Jérôme, Piguet, Françoise
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2023.107768