TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens

Targeting transforming growth factor-β (TGF-β) receptors is a promising pharmacological approach to normalize aberrant signaling in genetic and non-genetic TGF-β associated diseases including fibrosis, cancer, cardiovascular and musculoskeletal disorders. To identify novel TGF-β receptor kinase inhi...

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Main Authors: Marius Wits, Nicole Haarmans, Gonzalo Sanchez-Duffhues, Marie-José Goumans
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:SLAS Discovery
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Online Access:http://www.sciencedirect.com/science/article/pii/S2472555224000583
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author Marius Wits
Nicole Haarmans
Gonzalo Sanchez-Duffhues
Marie-José Goumans
author_facet Marius Wits
Nicole Haarmans
Gonzalo Sanchez-Duffhues
Marie-José Goumans
author_sort Marius Wits
collection DOAJ
description Targeting transforming growth factor-β (TGF-β) receptors is a promising pharmacological approach to normalize aberrant signaling in genetic and non-genetic TGF-β associated diseases including fibrosis, cancer, cardiovascular and musculoskeletal disorders. To identify novel TGF-β receptor kinase inhibitors, methods like in vitro kinase assays, western blot or transcriptional reporter assays are often used for screening purposes. While these methods may have certain advantages, the lack of integration of key features such as receptor specificity, high-throughput capability, and cellular context resemblance remains a major disadvantage. This deficiency could ultimately hinder the translation of study outcomes into later (clinical) stages of drug development. In this study, we introduce an adjusted and optimized live cell NanoBRET Target Engagement (TE)-based method to identify TGF-β receptor specific kinase inhibitors. This comprehensive toolkit contains various TGF-β type I and type II receptors, with corresponding nanoBRET tracers, and disease-related cell lines, including novel non-commercially available materials. The nanoBRET capacity and kinase inhibitory window can be significantly enhanced for functional measurements when stable expression cell lines and substantially low tracer concentrations are used. In addition, this system can be tailored to study TGF-β associated genetic disorders and possibly be used to screen for disease-specific therapeutics. Therefore, the use of this optimized, live cell, antibody-independent nanoBRET Target Engagement assay is highly encouraged for future high-throughput compound screens targeting TGF-β/BMP receptors.
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spelling doaj-art-26fa8b004f2a42ff99e746cff2cda2e02025-08-20T01:58:27ZengElsevierSLAS Discovery2472-55522024-12-0129810019610.1016/j.slasd.2024.100196TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screensMarius Wits0Nicole Haarmans1Gonzalo Sanchez-Duffhues2Marie-José Goumans3Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, the NetherlandsDepartment of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, the NetherlandsDepartment of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, the Netherlands; Nanomaterials and Nanotechnology Research Center (CINN), Spanish National Research Council (CSIC), Health Research Institute of Asturias (ISPA), 33011 Oviedo, Asturias, Spain; Corresponding authors at: Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, the Netherlands; Corresponding authors at: Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.Targeting transforming growth factor-β (TGF-β) receptors is a promising pharmacological approach to normalize aberrant signaling in genetic and non-genetic TGF-β associated diseases including fibrosis, cancer, cardiovascular and musculoskeletal disorders. To identify novel TGF-β receptor kinase inhibitors, methods like in vitro kinase assays, western blot or transcriptional reporter assays are often used for screening purposes. While these methods may have certain advantages, the lack of integration of key features such as receptor specificity, high-throughput capability, and cellular context resemblance remains a major disadvantage. This deficiency could ultimately hinder the translation of study outcomes into later (clinical) stages of drug development. In this study, we introduce an adjusted and optimized live cell NanoBRET Target Engagement (TE)-based method to identify TGF-β receptor specific kinase inhibitors. This comprehensive toolkit contains various TGF-β type I and type II receptors, with corresponding nanoBRET tracers, and disease-related cell lines, including novel non-commercially available materials. The nanoBRET capacity and kinase inhibitory window can be significantly enhanced for functional measurements when stable expression cell lines and substantially low tracer concentrations are used. In addition, this system can be tailored to study TGF-β associated genetic disorders and possibly be used to screen for disease-specific therapeutics. Therefore, the use of this optimized, live cell, antibody-independent nanoBRET Target Engagement assay is highly encouraged for future high-throughput compound screens targeting TGF-β/BMP receptors.http://www.sciencedirect.com/science/article/pii/S2472555224000583BMPDrug developmentRare diseaseCancerAngiogenesis
spellingShingle Marius Wits
Nicole Haarmans
Gonzalo Sanchez-Duffhues
Marie-José Goumans
TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens
SLAS Discovery
BMP
Drug development
Rare disease
Cancer
Angiogenesis
title TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens
title_full TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens
title_fullStr TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens
title_full_unstemmed TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens
title_short TGF-β receptor-specific NanoBRET Target Engagement in living cells for high-throughput kinase inhibitor screens
title_sort tgf β receptor specific nanobret target engagement in living cells for high throughput kinase inhibitor screens
topic BMP
Drug development
Rare disease
Cancer
Angiogenesis
url http://www.sciencedirect.com/science/article/pii/S2472555224000583
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AT gonzalosanchezduffhues tgfbreceptorspecificnanobrettargetengagementinlivingcellsforhighthroughputkinaseinhibitorscreens
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