Development and validation of an intravenous AAV gene therapy for mucopolysaccharidosis type IIIB in mouse and dog model of the pathology

{"title":"Development and validation of an intravenous AAV gene therapy for mucopolysaccharidosis type IIIB in mouse and dog model of the pathology","authors":"Elena Gaia Banchi , Nissrine Ballout , Rafael Alonso , Johan Deniaud , Sylvie Jacquot , Kevin Fransquin , Françoise Roux , Marie-Anne S. Colle , Jérôme Ausseil , Françoise Piguet","doi":"10.1016/j.ymgme.2024.108649","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108649"},"PeriodicalIF":3.7000,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular genetics and metabolism","FirstCategoryId":"99","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S109671922400533X","RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}