Frontiers in RNAi

Volume 1


Ralph A. Tripp, Jon M. Karpilow

DOI: 10.2174/97816080594091140101
eISBN: 978-1-60805-940-9, 2014
ISBN: 978-1-60805-941-6
ISSN: 2589-2932 (Print)
ISSN: 2352-8400 (Online)

Indexed in: EBSCO.

RNA Interference (RNAi) arrived in the scientific community just over a decade ago, and has since blossomed with new connections being...[view complete introduction]
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RNAi and Off-Target Effects

- Pp. 3-20 (18)

Amanda Birmingham, Andreas Kaufmann and Karol Kozak


RNA interference (RNAi) is widely used in high-throughput reverse genetics screens for basic research and drug discovery. However, this technique is known to produce “off-target” effects, or phenotypic results caused by an RNAi reagent’s knockdown of unintended genes rather than the gene of interest. Off-target effects are regulated through multiple mechanisms within the cell, and their presence can greatly complicate the interpretation of experimental data. We review the biology of offtargeting and discuss both bioinformatics and experimental approaches to reduce RNAi reagents’ off-target effects. Since such techniques cannot completely eliminate offtarget effects, we also discuss analysis methods developed to identify off-target effects in RNAi screening data and, in some cases, even leverage them to uncover novel biological functionality.

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