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Lipophilic siRNA conjugates yield durable silencing in extrahepatic tissues

Nature Biotechnology volume 40, pages 1439–1440 (2022)Cite this article

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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.

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Fig. 1: C16-siRNA distribution and activity in extrahepatic tissues.

References

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This is a summary of: Brown, K. M. et al. Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates. Nat. Biotechnol. https://doi.org/10.1038/s41587-022-01334-x (2022)

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Lipophilic siRNA conjugates yield durable silencing in extrahepatic tissues. Nat Biotechnol 40, 1439–1440 (2022). https://doi.org/10.1038/s41587-022-01335-w

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