Target protein identification in live cells and organisms with a non-diffusive proximity tagging system

Identifying target proteins for bioactive molecules is essential for understanding their mechanisms, developing improved derivatives, and minimizing off-target effects. Despite advances in target identification (target-ID) technologies, significant challenges remain, impeding drug development. Most...

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Main Authors: Yingjie Sun, Changheng Li, Xiaofei Deng, Wenjie Li, Xiaoyi Deng, Weiqi Ge, Miaoyuan Shi, Ying Guo, Yanxun V Yu, Hai-bing Zhou, Youngnam N Jin
Format: Article
Language:English
Published: eLife Sciences Publications Ltd 2024-12-01
Series:eLife
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Online Access:https://elifesciences.org/articles/102667
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author Yingjie Sun
Changheng Li
Xiaofei Deng
Wenjie Li
Xiaoyi Deng
Weiqi Ge
Miaoyuan Shi
Ying Guo
Yanxun V Yu
Hai-bing Zhou
Youngnam N Jin
author_facet Yingjie Sun
Changheng Li
Xiaofei Deng
Wenjie Li
Xiaoyi Deng
Weiqi Ge
Miaoyuan Shi
Ying Guo
Yanxun V Yu
Hai-bing Zhou
Youngnam N Jin
author_sort Yingjie Sun
collection DOAJ
description Identifying target proteins for bioactive molecules is essential for understanding their mechanisms, developing improved derivatives, and minimizing off-target effects. Despite advances in target identification (target-ID) technologies, significant challenges remain, impeding drug development. Most target-ID methods use cell lysates, but maintaining an intact cellular context is vital for capturing specific drug–protein interactions, such as those with transient protein complexes and membrane-associated proteins. To address these limitations, we developed POST-IT (Pup-On-target for Small molecule Target Identification Technology), a non-diffusive proximity tagging system for live cells, orthogonal to the eukaryotic system. POST-IT utilizes an engineered fusion of proteasomal accessory factor A and HaloTag to transfer Pup to proximal proteins upon directly binding to the small molecule. After significant optimization to eliminate self-pupylation and polypupylation, minimize depupylation, and optimize chemical linkers, POST-IT successfully identified known targets and discovered a new binder, SEPHS2, for dasatinib, and VPS37C as a new target for hydroxychloroquine, enhancing our understanding these drugs’ mechanisms of action. Furthermore, we demonstrated the application of POST-IT in live zebrafish embryos, highlighting its potential for broad biological research and drug development.
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spelling doaj-art-e3f6f4fa770347f5a71bc78b3411499a2025-08-20T02:39:40ZengeLife Sciences Publications LtdeLife2050-084X2024-12-011310.7554/eLife.102667Target protein identification in live cells and organisms with a non-diffusive proximity tagging systemYingjie Sun0Changheng Li1Xiaofei Deng2Wenjie Li3Xiaoyi Deng4Weiqi Ge5Miaoyuan Shi6Ying Guo7Yanxun V Yu8https://orcid.org/0000-0001-6617-0166Hai-bing Zhou9Youngnam N Jin10https://orcid.org/0009-0007-2632-3237Department of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Hematology, Zhongnan Hospital of Wuhan University, School of Pharmaceutical Sciences, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, China; Frontier Science Center for Immunology and Metabolism, Wuhan University, Wuhan, ChinaDepartment of Hematology, Zhongnan Hospital of Wuhan University, School of Pharmaceutical Sciences, Wuhan University, Wuhan, China; State Key Laboratory of Virology, Hubei Province Engineering and Technology Research Center for Fluorinated Pharmaceuticals, Key Laboratory of Combinatorial Biosynthesis and Drug Discovery (Wuhan University), Ministry of Education, Frontier Science Center for Immunology and Metabolism, Wuhan University, Wuhan, ChinaDepartment of Neurology, Medical Research Institute, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, China; Frontier Science Center for Immunology and Metabolism, Wuhan University, Wuhan, ChinaIdentifying target proteins for bioactive molecules is essential for understanding their mechanisms, developing improved derivatives, and minimizing off-target effects. Despite advances in target identification (target-ID) technologies, significant challenges remain, impeding drug development. Most target-ID methods use cell lysates, but maintaining an intact cellular context is vital for capturing specific drug–protein interactions, such as those with transient protein complexes and membrane-associated proteins. To address these limitations, we developed POST-IT (Pup-On-target for Small molecule Target Identification Technology), a non-diffusive proximity tagging system for live cells, orthogonal to the eukaryotic system. POST-IT utilizes an engineered fusion of proteasomal accessory factor A and HaloTag to transfer Pup to proximal proteins upon directly binding to the small molecule. After significant optimization to eliminate self-pupylation and polypupylation, minimize depupylation, and optimize chemical linkers, POST-IT successfully identified known targets and discovered a new binder, SEPHS2, for dasatinib, and VPS37C as a new target for hydroxychloroquine, enhancing our understanding these drugs’ mechanisms of action. Furthermore, we demonstrated the application of POST-IT in live zebrafish embryos, highlighting its potential for broad biological research and drug development.https://elifesciences.org/articles/102667proximity labelingPafAdasatinibchloroquinezebrafishtarget identification
spellingShingle Yingjie Sun
Changheng Li
Xiaofei Deng
Wenjie Li
Xiaoyi Deng
Weiqi Ge
Miaoyuan Shi
Ying Guo
Yanxun V Yu
Hai-bing Zhou
Youngnam N Jin
Target protein identification in live cells and organisms with a non-diffusive proximity tagging system
eLife
proximity labeling
PafA
dasatinib
chloroquine
zebrafish
target identification
title Target protein identification in live cells and organisms with a non-diffusive proximity tagging system
title_full Target protein identification in live cells and organisms with a non-diffusive proximity tagging system
title_fullStr Target protein identification in live cells and organisms with a non-diffusive proximity tagging system
title_full_unstemmed Target protein identification in live cells and organisms with a non-diffusive proximity tagging system
title_short Target protein identification in live cells and organisms with a non-diffusive proximity tagging system
title_sort target protein identification in live cells and organisms with a non diffusive proximity tagging system
topic proximity labeling
PafA
dasatinib
chloroquine
zebrafish
target identification
url https://elifesciences.org/articles/102667
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