{"title":"心脏再生的可调基因疗法。","authors":"Gabriele D’Uva","doi":"10.1038/s44161-025-00666-6","DOIUrl":null,"url":null,"abstract":"AAV-based gene therapies hold promise for treating disease, but their long-lasting gene expression limits their use in regenerative medicine. A study now presents DreAM, a drug-inducible AAV system that enables tunable tissue-specific gene activation via the splicing modulator risdiplam, and its potential application in myocardial infarction.","PeriodicalId":74245,"journal":{"name":"Nature cardiovascular research","volume":"4 7","pages":"794-796"},"PeriodicalIF":10.8000,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A tunable gene therapy for heart regeneration\",\"authors\":\"Gabriele D’Uva\",\"doi\":\"10.1038/s44161-025-00666-6\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"AAV-based gene therapies hold promise for treating disease, but their long-lasting gene expression limits their use in regenerative medicine. A study now presents DreAM, a drug-inducible AAV system that enables tunable tissue-specific gene activation via the splicing modulator risdiplam, and its potential application in myocardial infarction.\",\"PeriodicalId\":74245,\"journal\":{\"name\":\"Nature cardiovascular research\",\"volume\":\"4 7\",\"pages\":\"794-796\"},\"PeriodicalIF\":10.8000,\"publicationDate\":\"2025-06-13\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Nature cardiovascular research\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.nature.com/articles/s44161-025-00666-6\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CARDIAC & CARDIOVASCULAR SYSTEMS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nature cardiovascular research","FirstCategoryId":"1085","ListUrlMain":"https://www.nature.com/articles/s44161-025-00666-6","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
AAV-based gene therapies hold promise for treating disease, but their long-lasting gene expression limits their use in regenerative medicine. A study now presents DreAM, a drug-inducible AAV system that enables tunable tissue-specific gene activation via the splicing modulator risdiplam, and its potential application in myocardial infarction.
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