研究人员为提升 ssODN 基因编辑效率，研究 LNA 修饰及链长影响，发现优化方法且该法对碱基替换有效。 在生物技术蓬勃发展的当下，基因组编辑就像一把神奇的 “基因剪刀”，能精准地修改生物的遗传密码，在高效生物质生产和疾病治疗等领域发挥着关键作用。

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The study presents valuable findings regarding the incidence and clinical impact of a mutation in a cardiac muscle protein and its association with the development of atrial fibrillation. The authors ...

Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. In this study, we analyze the long noncoding ...

Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. An evolutionarily conserved micropeptide, ...

UCART22 Phase 1 dataset and late-stage development strategy expected in the third quarter of 2025; Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) granted by FDA and ODD ...

The Ken & Ruth Davee Department of Neurology, Feinberg School of Medicine, Northwestern University, Chicago, United States Simpson Querrey Institute, Northwestern University, Chicago, United States ...