eLifeGeneticsPeer-reviewed journal article
Precision RNAi using synthetic shRNAmir target sites
Thomas HoffmanniD, Alexandra Hörmann, Maja Corcokovic, Jakub ZmajkoviciD, Matthias HinterndorferiD, Jasko Salkanovic, Fiona SpreitzeriD, Anna KöferleiD, Katrin Gitschtaler, Alexandra PopaiD, Sarah Oberndorfer, Florian AnderschiD, Markus Schaefer, Michaela Fellner, Nicole BudanoiD, Jan G. RuppertiD, Paolo ChettaiD, Melanie WurmiD, Johannes A. ZuberiD, Ralph A. NeumülleriD
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 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.
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