{"title":"按指令设计定量基因治疗。","authors":"Connie An, Noa Katz, Xiaojing J Gao","doi":"10.1016/j.cels.2025.101323","DOIUrl":null,"url":null,"abstract":"<p><p>Gene replacement therapies can generate unnaturally high levels of transgene expression, potentially compromising their safety or efficacy. Variable gene delivery compounds this problem, leading to heterogeneous expression. To address this limitation, ComMAND, a microRNA-based biomolecular circuit assisted by computational models, reduces cell-to-cell variation in gene expression.</p>","PeriodicalId":93929,"journal":{"name":"Cell systems","volume":"16 6","pages":"101323"},"PeriodicalIF":7.7000,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Designing quantitative gene therapy on ComMAND.\",\"authors\":\"Connie An, Noa Katz, Xiaojing J Gao\",\"doi\":\"10.1016/j.cels.2025.101323\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Gene replacement therapies can generate unnaturally high levels of transgene expression, potentially compromising their safety or efficacy. Variable gene delivery compounds this problem, leading to heterogeneous expression. To address this limitation, ComMAND, a microRNA-based biomolecular circuit assisted by computational models, reduces cell-to-cell variation in gene expression.</p>\",\"PeriodicalId\":93929,\"journal\":{\"name\":\"Cell systems\",\"volume\":\"16 6\",\"pages\":\"101323\"},\"PeriodicalIF\":7.7000,\"publicationDate\":\"2025-06-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cell systems\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.cels.2025.101323\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cell systems","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.cels.2025.101323","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Gene replacement therapies can generate unnaturally high levels of transgene expression, potentially compromising their safety or efficacy. Variable gene delivery compounds this problem, leading to heterogeneous expression. To address this limitation, ComMAND, a microRNA-based biomolecular circuit assisted by computational models, reduces cell-to-cell variation in gene expression.
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