Precision RNAi using synthetic shRNAmir target sites

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...

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Autores principales: Hoffmann, Thomas, Hörmann, Alexandra, Corcokovic, Maja, Zmajkovic, Jakub, Hinterndorfer, Matthias, Salkanovic, Jasko, Spreitzer, Fiona, Köferle, Anna, Gitschtaler, Katrin, Popa, Alexandra, Oberndorfer, Sarah, Andersch, Florian, Schaefer, Markus, Fellner, Michaela, Budano, Nicole, Ruppert, Jan G, Chetta, Paolo, Wurm, Melanie, Zuber, Johannes, Neumüller, Ralph A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: eLife Sciences Publications, Ltd 2023
Materias:
Cancer Biology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10409502/
https://www.ncbi.nlm.nih.gov/pubmed/37552050
http://dx.doi.org/10.7554/eLife.84792
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author Hoffmann, Thomas
Hörmann, Alexandra
Corcokovic, Maja
Zmajkovic, Jakub
Hinterndorfer, Matthias
Salkanovic, Jasko
Spreitzer, Fiona
Köferle, Anna
Gitschtaler, Katrin
Popa, Alexandra
Oberndorfer, Sarah
Andersch, Florian
Schaefer, Markus
Fellner, Michaela
Budano, Nicole
Ruppert, Jan G
Chetta, Paolo
Wurm, Melanie
Zuber, Johannes
Neumüller, Ralph A
author_facet Hoffmann, Thomas
Hörmann, Alexandra
Corcokovic, Maja
Zmajkovic, Jakub
Hinterndorfer, Matthias
Salkanovic, Jasko
Spreitzer, Fiona
Köferle, Anna
Gitschtaler, Katrin
Popa, Alexandra
Oberndorfer, Sarah
Andersch, Florian
Schaefer, Markus
Fellner, Michaela
Budano, Nicole
Ruppert, Jan G
Chetta, Paolo
Wurm, Melanie
Zuber, Johannes
Neumüller, Ralph A
author_sort Hoffmann, Thomas
collection PubMed
description 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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spelling pubmed-104095022023-08-09 Precision RNAi using synthetic shRNAmir target sites Hoffmann, Thomas Hörmann, Alexandra Corcokovic, Maja Zmajkovic, Jakub Hinterndorfer, Matthias Salkanovic, Jasko Spreitzer, Fiona Köferle, Anna Gitschtaler, Katrin Popa, Alexandra Oberndorfer, Sarah Andersch, Florian Schaefer, Markus Fellner, Michaela Budano, Nicole Ruppert, Jan G Chetta, Paolo Wurm, Melanie Zuber, Johannes Neumüller, Ralph A eLife Cancer Biology 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. eLife Sciences Publications, Ltd 2023-08-08 /pmc/articles/PMC10409502/ /pubmed/37552050 http://dx.doi.org/10.7554/eLife.84792 Text en © 2023, Hoffmann, Hörmann, Corcokovic et al https://creativecommons.org/licenses/by/4.0/This article is distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use and redistribution provided that the original author and source are credited.
spellingShingle Cancer Biology
Hoffmann, Thomas
Hörmann, Alexandra
Corcokovic, Maja
Zmajkovic, Jakub
Hinterndorfer, Matthias
Salkanovic, Jasko
Spreitzer, Fiona
Köferle, Anna
Gitschtaler, Katrin
Popa, Alexandra
Oberndorfer, Sarah
Andersch, Florian
Schaefer, Markus
Fellner, Michaela
Budano, Nicole
Ruppert, Jan G
Chetta, Paolo
Wurm, Melanie
Zuber, Johannes
Neumüller, Ralph A
Precision RNAi using synthetic shRNAmir target sites
title Precision RNAi using synthetic shRNAmir target sites
title_full Precision RNAi using synthetic shRNAmir target sites
title_fullStr Precision RNAi using synthetic shRNAmir target sites
title_full_unstemmed Precision RNAi using synthetic shRNAmir target sites
title_short Precision RNAi using synthetic shRNAmir target sites
title_sort precision rnai using synthetic shrnamir target sites
topic Cancer Biology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10409502/
https://www.ncbi.nlm.nih.gov/pubmed/37552050
http://dx.doi.org/10.7554/eLife.84792
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