{"title":"优化等位基因特异性沉默显性阴性 COL6A1 G293R 取代导致胶原 VI 相关营养不良症","authors":"Astrid Brull, A. Sarathy, Véronique Bolduc, Grace S. Chen, R. McCarty, Carsten G. Bönnemann","doi":"10.1016/j.omtn.2024.102178","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"17 11","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy\",\"authors\":\"Astrid Brull, A. Sarathy, Véronique Bolduc, Grace S. Chen, R. McCarty, Carsten G. Bönnemann\",\"doi\":\"10.1016/j.omtn.2024.102178\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":503104,\"journal\":{\"name\":\"Molecular Therapy - Nucleic Acids\",\"volume\":\"17 11\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-03-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Therapy - Nucleic Acids\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.omtn.2024.102178\",\"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 - Nucleic Acids","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.omtn.2024.102178","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
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