1. Precision RNAi using synthetic shRNAmir target sites
- Author
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Thomas Hoffmann, Alexandra Hörmann, Maja Corcokovic, Jakub Zmajkovic, Matthias Hinterndorfer, Jasko Salkanovic, Fiona Spreitzer, Anna Köferle, Katrin Gitschtaler, Alexandra Popa, Sarah Oberndorfer, Florian Andersch, Markus Schäfer, Michaela Fellner, Nicole Budano, Jan G. Ruppert, Paolo Chetta, Melanie Wurm, Johannes Zuber, and Ralph A. Neumüller
- Abstract
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, inducible, and reversible 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.
- Published
- 2023
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