Precision RNAi using synthetic shRNAmir target sites.

Autor:	Hoffmann T; 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., Hörmann A; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Corcokovic M; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Zmajkovic J; Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria., Hinterndorfer M; Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria., Salkanovic J; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Spreitzer F; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Köferle A; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Gitschtaler K; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Popa A; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Oberndorfer S; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Andersch F; Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria., Schaefer M; Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria., Fellner M; Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria., Budano N; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Ruppert JG; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Chetta P; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Wurm M; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria., Zuber J; Research Institute of Molecular Pathology (IMP), Campus-Vienna-Biocenter 1, Vienna, Austria.; Medical University of Vienna, Vienna BioCenter, Vienna, Austria., Neumüller RA; Boehringer Ingelheim RCV GmbH & Co KG, Doktor-Boehringer-Gasse, Vienna, Austria.
Jazyk:	angličtina
Zdroj:	ELife [Elife] 2023 Aug 08; Vol. 12. Date of Electronic Publication: 2023 Aug 08.
DOI:	10.7554/eLife.84792
Abstrakt:	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. Competing Interests: TH Full-time employee of Advantage Therapeutics GmbH, AH, MC, JS, FS, AK, KG, AP, SO, NB, JR, PC, MW, RN Full time employee Boehringer Ingelheim, JZ, MH, FA, MS, MF, JZ No competing interests declared (© 2023, Hoffmann, Hörmann, Corcokovic et al.)
Databáze:	MEDLINE
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