RNAi in Cell Nuclei: Potential for a new layer of biological regulation and a new strategy for therapeutic discovery
- ↵* Corresponding author; email: david.corey{at}utsouthwestern.edu
Abstract
RNAi interference (RNAi) is almost always associated with inhibition of translation in cell cytoplasm. MicroRNAs (miRNAs) and critical RNAi protein factors like argonaute (AGO) and trinucleotide repeat binding containing 6 protein (TNRC6), however, are also found in cell nuclei, suggesting that nuclear miRNAs may be targets for gene regulation. Designed small duplex RNAs (dsRNAs) can modulate nuclear processes such as transcription and splicing, suggesting that they can also provide leads for therapeutic discovery. The goal of this perspective is to provide the background on nuclear RNAi necessary to guide discussions on whether nuclear RNAi can play a role in therapeutic development programs.
Keywords
- Received November 1, 2022.
- Accepted January 9, 2023.
- Published by Cold Spring Harbor Laboratory Press for the RNA Society
This article, published in RNA, is available under a Creative Commons License (Attribution-NonCommercial 4.0 International), as described at http://creativecommons.org/licenses/by-nc/4.0/.
