Arunabha Ghosh, Bénédicte Héron, Aimee Donald, Harriet Chang, Laura Furness, Nina Movsesyan, Claudia Ravelli, Simon Jones, Raymond Y. Wang
{"title":"Safety and preliminary efficacy of LYS-GM101 gene therapy in patients with GM1 gangliosidosis: Results of a phase I/II open-label clinical trial","authors":"Arunabha Ghosh, Bénédicte Héron, Aimee Donald, Harriet Chang, Laura Furness, Nina Movsesyan, Claudia Ravelli, Simon Jones, Raymond Y. Wang","doi":"10.1016/j.ymgme.2023.107839","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":504050,"journal":{"name":"Molecular Genetics and Metabolism","volume":"45 23","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Genetics and Metabolism","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.ymgme.2023.107839","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}