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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References
Setten, R. L., Rossi, J. J. & Han, S. P. The current state and future directions of RNAi-based therapeutics. Nat. Rev. Drug Discov. 18, 421–446 (2019). A Review article that summarizes the RNAi therapeutics field.
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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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DOI: https://doi.org/10.1038/s41587-022-01335-w
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