Precision RNAi using synthetic shRNAmir target sites

Loss-of-function genetic tools are widely applied for validating therapeutic targets, but their utility remains limited by incomplete on- and uncontrolled off-target effects. We describe artificial RNA interference (ARTi) based on synthetic, ultra-potent, off-target-free shRNAs that enable efficient...

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Published ineLife Vol. 12
Main Authors Hoffmann, Thomas, Hörmann, Alexandra, Corcokovic, Maja, Zmajkovic, Jakub, Hinterndorfer, Matthias, Salkanovic, Jasko, Spreitzer, Fiona, Köferle, Anna, Gitschtaler, Katrin, Popa, Alexandra, Oberndorfer, Sarah, Andersch, Florian, Schaefer, Markus, Fellner, Michaela, Budano, Nicole, Ruppert, Jan G, Chetta, Paolo, Wurm, Melanie, Zuber, Johannes, Neumüller, Ralph A
Format Journal Article
LanguageEnglish
Published 08.08.2023
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Summary:Loss-of-function genetic tools are widely applied for validating therapeutic targets, but their utility remains limited by incomplete on- and uncontrolled off-target effects. We describe artificial RNA interference (ARTi) based on synthetic, ultra-potent, off-target-free shRNAs that enable efficient and inducible suppression of any gene upon introduction of a synthetic target sequence into non-coding transcript regions. ARTi establishes a scalable loss-of-function tool with full control over on- and off-target effects.
ISSN:2050-084X
2050-084X
DOI:10.7554/eLife.84792.3