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Volume 40 Issue 10, October 2022

Extrahepatic delivery of RNA therapeutics

A model of 2&#x2032-O-hexadecyl (C16)-conjugated siRNA (orange) in complex with RISC (white) and mRNA (green). Brown et al. show that C16-conjugated siRNA achieves efficient, durable targeted gene silencing in the central nervous system, eye and lung.

See Brown et al.

Image: Alnylam Pharmaceuticals Inc. Cover Design: Erin Dewalt.

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    • The COVID-19 pandemic dispelled some myths underlying intellectual property policy and revealed how stakeholders can develop policies to accelerate development and ensure access using existing tools and experimenting with open science.

      • E. Richard Gold
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    • Recent patents relating to methods and devices for optogenetic control of cells.

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  • Are major decisions in the cell made through pairs of interacting loci (enhancers and promoters) or larger teams of cooperating regulatory elements? A new genome-wide assay and algorithm answers this question and provides a scalable technology to link new dimensions of genome structure with cellular function.

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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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