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.
Keywords: EGFR; KRAS; RNAi; cancer; cancer biology; human; loss-of-function; mouse; target validation.
© 2023, Hoffmann, Hörmann, Corcokovic et al.