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Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors

Attenuating aberrant transcriptional circuits holds great promise for the treatment of numerous diseases, including cancer. However, development of transcriptional inhibitors is hampered by the lack of a generally accepted functional cellular readout to characterize their target specificity and on-t...

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Autores principales: Harada, Taku, Perez, Monika W., Kalfon, Jérémie, Braes, Flora Dievenich, Batley, Rashad, Eagle, Kenneth, Nabet, Behnam, Leifer, Becky, Kruell, Jasmin, Paralkar, Vikram R., Stegmaier, Kimberly, Koehler, Angela N., Orkin, Stuart H., Pimkin, Maxim
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10104119/
https://www.ncbi.nlm.nih.gov/pubmed/37066194
http://dx.doi.org/10.1101/2023.04.07.536032
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author Harada, Taku
Perez, Monika W.
Kalfon, Jérémie
Braes, Flora Dievenich
Batley, Rashad
Eagle, Kenneth
Nabet, Behnam
Leifer, Becky
Kruell, Jasmin
Paralkar, Vikram R.
Stegmaier, Kimberly
Koehler, Angela N.
Orkin, Stuart H.
Pimkin, Maxim
author_facet Harada, Taku
Perez, Monika W.
Kalfon, Jérémie
Braes, Flora Dievenich
Batley, Rashad
Eagle, Kenneth
Nabet, Behnam
Leifer, Becky
Kruell, Jasmin
Paralkar, Vikram R.
Stegmaier, Kimberly
Koehler, Angela N.
Orkin, Stuart H.
Pimkin, Maxim
author_sort Harada, Taku
collection PubMed
description Attenuating aberrant transcriptional circuits holds great promise for the treatment of numerous diseases, including cancer. However, development of transcriptional inhibitors is hampered by the lack of a generally accepted functional cellular readout to characterize their target specificity and on-target activity. We benchmarked the direct gene-regulatory signatures of six agents reported as inhibitors of the oncogenic transcription factor MYB against targeted MYB degradation in a nascent transcriptomics assay. The inhibitors demonstrated partial specificity for MYB target genes but displayed significant off-target activity. Unexpectedly, the inhibitors displayed bimodal on-target effects, acting as mixed agonists-antagonists. Our data uncover unforeseen agonist effects of small molecules originally developed as TF inhibitors and argue that rapid-kinetics benchmarking against degron models should be used for functional characterization of transcriptional modulators.
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spelling pubmed-101041192023-04-15 Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors Harada, Taku Perez, Monika W. Kalfon, Jérémie Braes, Flora Dievenich Batley, Rashad Eagle, Kenneth Nabet, Behnam Leifer, Becky Kruell, Jasmin Paralkar, Vikram R. Stegmaier, Kimberly Koehler, Angela N. Orkin, Stuart H. Pimkin, Maxim bioRxiv Article Attenuating aberrant transcriptional circuits holds great promise for the treatment of numerous diseases, including cancer. However, development of transcriptional inhibitors is hampered by the lack of a generally accepted functional cellular readout to characterize their target specificity and on-target activity. We benchmarked the direct gene-regulatory signatures of six agents reported as inhibitors of the oncogenic transcription factor MYB against targeted MYB degradation in a nascent transcriptomics assay. The inhibitors demonstrated partial specificity for MYB target genes but displayed significant off-target activity. Unexpectedly, the inhibitors displayed bimodal on-target effects, acting as mixed agonists-antagonists. Our data uncover unforeseen agonist effects of small molecules originally developed as TF inhibitors and argue that rapid-kinetics benchmarking against degron models should be used for functional characterization of transcriptional modulators. Cold Spring Harbor Laboratory 2023-04-07 /pmc/articles/PMC10104119/ /pubmed/37066194 http://dx.doi.org/10.1101/2023.04.07.536032 Text en https://creativecommons.org/licenses/by-nc-nd/4.0/This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (https://creativecommons.org/licenses/by-nc-nd/4.0/) , which allows reusers to copy and distribute the material in any medium or format in unadapted form only, for noncommercial purposes only, and only so long as attribution is given to the creator.
spellingShingle Article
Harada, Taku
Perez, Monika W.
Kalfon, Jérémie
Braes, Flora Dievenich
Batley, Rashad
Eagle, Kenneth
Nabet, Behnam
Leifer, Becky
Kruell, Jasmin
Paralkar, Vikram R.
Stegmaier, Kimberly
Koehler, Angela N.
Orkin, Stuart H.
Pimkin, Maxim
Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors
title Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors
title_full Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors
title_fullStr Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors
title_full_unstemmed Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors
title_short Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors
title_sort rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of myb inhibitors
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10104119/
https://www.ncbi.nlm.nih.gov/pubmed/37066194
http://dx.doi.org/10.1101/2023.04.07.536032
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