Precision RNAi using synthetic shRNAmir target sites

Autor: 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 Schaefer, Michaela Fellner, Nicole Budano, Jan G Ruppert, Paolo Chetta, Melanie Wurm, Johannes Zuber, Ralph A Neumüller
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: eLife, Vol 12 (2023)
Druh dokumentu: article
ISSN: 2050-084X
DOI: 10.7554/eLife.84792
Popis: 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.
Databáze: Directory of Open Access Journals