基因治疗的未来：更安全的载体，更清晰的焦点：引人注目的失败需要深入的根本原因分析，但如果该领域愿意学习和发展，AAV的变革潜力仍然是可以实现的。

{"title":"基因治疗的未来：更安全的载体，更清晰的焦点：引人注目的失败需要深入的根本原因分析，但如果该领域愿意学习和发展，AAV的变革潜力仍然是可以实现的。","authors":"Jeffrey S Chamberlain,Beverly L Davidson,Lindsey A George,Barry J Byrne,David Barrett","doi":"10.1016/j.ymthe.2025.08.029","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"77 1","pages":""},"PeriodicalIF":12.0000,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve.\",\"authors\":\"Jeffrey S Chamberlain,Beverly L Davidson,Lindsey A George,Barry J Byrne,David Barrett\",\"doi\":\"10.1016/j.ymthe.2025.08.029\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":19020,\"journal\":{\"name\":\"Molecular Therapy\",\"volume\":\"77 1\",\"pages\":\"\"},\"PeriodicalIF\":12.0000,\"publicationDate\":\"2025-09-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1016/j.ymthe.2025.08.029\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"BIOTECHNOLOGY & APPLIED MICROBIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.ymthe.2025.08.029","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}