在r6/2亨廷顿氏病转基因小鼠模型中，皮下给予patrol™研究性基因治疗后，整个大脑中突变htt转录的等位基因选择性抑制

{"title":"在r6/2亨廷顿氏病转基因小鼠模型中，皮下给予patrol™研究性基因治疗后，整个大脑中突变htt转录的等位基因选择性抑制","authors":"R. Friedlander, V. Caro, D. Carlisle, N. Monks, Dani M. Stoltzfus, Natasha L Bennett, Y. Suofu, Jinho Kim, Fang Li, Xiaomin Wang, Stacy Yeh, O. V. Baranov, S. Baranov, Barry A. Badeau, Jeremy Gleaton, Gabriel A Cintrón-Rosado, Ryan R Dyer, D. Stephan, W. R. Mann, Sandra Rojas-Caro","doi":"10.1136/jnnp-2022-ehdn.232","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"1 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"I06 Allele-selective inhibition of mutant htt transcription throughout the brain after subcutaneous administration of a patrol™-enabled investigational genetic therapy in the r6/2 Huntington’s disease transgenic mouse model\",\"authors\":\"R. Friedlander, V. Caro, D. Carlisle, N. Monks, Dani M. Stoltzfus, Natasha L Bennett, Y. Suofu, Jinho Kim, Fang Li, Xiaomin Wang, Stacy Yeh, O. V. Baranov, S. Baranov, Barry A. Badeau, Jeremy Gleaton, Gabriel A Cintrón-Rosado, Ryan R Dyer, D. Stephan, W. R. Mann, Sandra Rojas-Caro\",\"doi\":\"10.1136/jnnp-2022-ehdn.232\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":444837,\"journal\":{\"name\":\"I: Experimental therapeutics – preclinical\",\"volume\":\"1 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2022-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"I: Experimental therapeutics – preclinical\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1136/jnnp-2022-ehdn.232\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"I: Experimental therapeutics – preclinical","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1136/jnnp-2022-ehdn.232","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}