在IV rAAV9-hNAGLU基因递送后，MPS IIIB小鼠的代谢组学损伤几乎完全纠正，相应的功能获益

{"title":"在IV rAAV9-hNAGLU基因递送后，MPS IIIB小鼠的代谢组学损伤几乎完全纠正，相应的功能获益","authors":"H. Fu, Aaron S. Meadows, Tierra A Ware, R. Mohney, D. Mccarty","doi":"10.1016/j.ymthe.2016.12.025","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"22 1","pages":"792-802"},"PeriodicalIF":0.0000,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"15","resultStr":"{\"title\":\"Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.\",\"authors\":\"H. Fu, Aaron S. Meadows, Tierra A Ware, R. Mohney, D. Mccarty\",\"doi\":\"10.1016/j.ymthe.2016.12.025\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":19039,\"journal\":{\"name\":\"Molecular Therapy: the Journal of the American Society of Gene Therapy\",\"volume\":\"22 1\",\"pages\":\"792-802\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2017-03-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"15\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Therapy: the Journal of the American Society of Gene Therapy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.ymthe.2016.12.025\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Therapy: the Journal of the American Society of Gene Therapy","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.ymthe.2016.12.025","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}