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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
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
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。