eLife (Aug 2023)
Precision RNAi using synthetic shRNAmir target sites
- 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
Affiliations
- Thomas Hoffmann
- ORCiD
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria; Vienna BioCenter PhD Program, Doctoral School of the University at Vienna and Medical University of Vienna, Vienna BioCenter (VBC), Vienna, Austria
- Alexandra Hörmann
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Maja Corcokovic
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Jakub Zmajkovic
- ORCiD
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria
- Matthias Hinterndorfer
- ORCiD
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria
- Jasko Salkanovic
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Fiona Spreitzer
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Anna Köferle
- ORCiD
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Katrin Gitschtaler
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Alexandra Popa
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Sarah Oberndorfer
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Florian Andersch
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria
- Markus Schaefer
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria
- Michaela Fellner
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria
- Nicole Budano
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Jan G Ruppert
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Paolo Chetta
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Melanie Wurm
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- Johannes Zuber
- ORCiD
- Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria; Medical University of Vienna, Vienna BioCenter, Vienna, Austria
- Ralph A Neumüller
- ORCiD
- Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria
- DOI
- https://doi.org/10.7554/eLife.84792
- Journal volume & issue
-
Vol. 12
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.
Keywords
