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A multiplexed time-resolved fluorescence resonance energy transfer ultrahigh-throughput screening assay for targeting the SMAD4–SMAD3–DNA complex
Wukun Ouyang1 , Qianjin Li1 , Qiankun Niu1 , Min Qui1,2 , Haian Fu1,2,3 , Yuhong Du1,2,* , Xiulei Mo1,*
1Department of Pharmacology and Chemical Biology, Emory University School of Medicine, Atlanta, GA 30322, USA
2Emory Chemical Biology Discovery Center, Emory University School of Medicine, Atlanta, GA 30322, USA
3Department of Hematology and Medical Oncology and Winship Cancer Institute, Emory University, Atlanta, GA 30322, USA
*Correspondence to:Yuhong Du , Email:dyuhong@emory.edu Xiulei Mo , Email:XMO@emory.edu
J Mol Cell Biol, Volume 15, Issue 11, November 2023, mjad068,  https://doi.org/10.1093/jmcb/mjad068
Keyword: TGFβ/SMAD4 signaling, high-throughput screening, TR-FRET

The transforming growth factor-beta (TGFβ) signaling pathway plays crucial roles in the establishment of an immunosuppressive tumor microenvironment, making anti-TGFβ agents a significant area of interest in cancer immunotherapy. However, the clinical translation of current anti-TGFβ agents that target upstream cytokines and receptors remains challenging. Therefore, the development of small-molecule inhibitors specifically targeting SMAD4, the downstream master regulator of the TGFβ pathway, would offer an alternative approach with significant therapeutic potential for anti-TGFβ signaling. In this study, we present the development of a cell lysate-based multiplexed time-resolved fluorescence resonance energy transfer (TR-FRET) assay in an ultrahigh-throughput screening (uHTS) 1536-well plate format. This assay enables simultaneous monitoring of the protein‒protein interaction between SMAD4 and SMAD3, as well as the protein‒DNA interaction between SMADs and their consensus DNA-binding motif. The multiplexed TR-FRET assay exhibits high sensitivity, allowing the dynamic analysis of the SMAD4–SMAD3–DNA complex at single-amino acid resolution. Moreover, the multiplexed uHTS assay demonstrates robustness for screening small-molecule inhibitors. Through a pilot screening of an FDA-approved bioactive compound library, we identified gambogic acid and gambogenic acid as potential hit compounds. These proof-of-concept findings underscore the utility of our optimized multiplexed TR-FRET platform for large-scale screening to discover small-molecule inhibitors that target the SMAD4–SMAD3–DNA complex as novel anti-TGFβ signaling agents.