当前位置: X-MOL 学术Nat. Biotechnol. › 论文详情
Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)
Lipophilic siRNA conjugates yield durable silencing in extrahepatic tissues
Nature Biotechnology ( IF 46.9 ) Pub Date : 2022-06-08 , DOI: 10.1038/s41587-022-01335-w


RNA interference (RNAi) therapeutics can silence disease-causing gene transcripts, but extrahepatic delivery has been challenging. Conjugating short interfering RNAs (siRNAs) to a lipophilic alkyl chain enabled safe delivery and long-term mRNA silencing in the brain, eye and lung in animal models, thereby opening new applications for RNAi therapeutics.

中文翻译:

亲脂性 siRNA 偶联物在肝外组织中产生持久的沉默

RNA 干扰 (RNAi) 疗法可以沉默致病基因转录物,但肝外递送一直具有挑战性。将短干扰 RNA (siRNA) 与亲脂性烷基链结合,可以在动物模型的大脑、眼睛和肺中实现安全递送和长期 mRNA 沉默,从而为 RNAi 疗法开辟了新的应用。
更新日期:2022-06-08
down
wechat
bug