GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins

Membrane protein degradation is a cutting-edge field in targeted protein degradation (TPD). Herein, we developed glypican-3 (GPC3)-mediated lysosome-targeting chimeras (GLTACs) as a novel strategy for the targeted degradation of tumor-specific membrane proteins. GLTACs utilize tumor-specific express...

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Main Authors: Yuxin Fang, Yaojin Zhu, Wei Wang, Zhewei Xia, Shipeng He, Guoqiang Dong, Chunquan Sheng
Format: Article
Language:English
Published: Elsevier 2025-04-01
Series:Acta Pharmaceutica Sinica B
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Online Access:http://www.sciencedirect.com/science/article/pii/S2211383525001339
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author Yuxin Fang
Yaojin Zhu
Wei Wang
Zhewei Xia
Shipeng He
Guoqiang Dong
Chunquan Sheng
author_facet Yuxin Fang
Yaojin Zhu
Wei Wang
Zhewei Xia
Shipeng He
Guoqiang Dong
Chunquan Sheng
author_sort Yuxin Fang
collection DOAJ
description Membrane protein degradation is a cutting-edge field in targeted protein degradation (TPD). Herein, we developed glypican-3 (GPC3)-mediated lysosome-targeting chimeras (GLTACs) as a novel strategy for the targeted degradation of tumor-specific membrane proteins. GLTACs utilize tumor-specific expression and endocytosis properties of GPC3 to degrade membrane proteins. By conjugating a GPC3-targeting peptide with the ligand of protein of interest (POI), GLTACs induce the formation of a ternary complex that is internalized into lysosomes, leading to the degradation of the POI. The effectiveness and specificity of GLTACs were validated by designing PD-L1, c-Met, and FGFR1 degraders. In particular, GLTAC WP0 potently degraded PD-L1 and induced T-cell-mediated tumor killing against HepG2 cells, highlighting the potential therapeutic applications. The development of GLTAC technology expands the scope of TPD strategies and opens new avenues for discovering novel therapeutic modalities against challenging protein targets.
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issn 2211-3835
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publishDate 2025-04-01
publisher Elsevier
record_format Article
series Acta Pharmaceutica Sinica B
spelling doaj-art-79c1d75a3a1f4203be9961da3cd9d7d12025-08-20T01:51:13ZengElsevierActa Pharmaceutica Sinica B2211-38352025-04-011542156216910.1016/j.apsb.2025.02.037GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteinsYuxin Fang0Yaojin Zhu1Wei Wang2Zhewei Xia3Shipeng He4Guoqiang Dong5Chunquan Sheng6Center for Basic Research and Innovation of Medicine and Pharmacy (MOE), School of Pharmacy, Second Military Medical University (Naval Medical University), Shanghai 200433, ChinaInstitute of Translational Medicine, Shanghai University, Shanghai 200444, ChinaCenter for Basic Research and Innovation of Medicine and Pharmacy (MOE), School of Pharmacy, Second Military Medical University (Naval Medical University), Shanghai 200433, ChinaCenter for Basic Research and Innovation of Medicine and Pharmacy (MOE), School of Pharmacy, Second Military Medical University (Naval Medical University), Shanghai 200433, ChinaInstitute of Translational Medicine, Shanghai University, Shanghai 200444, China; Corresponding authors.Center for Basic Research and Innovation of Medicine and Pharmacy (MOE), School of Pharmacy, Second Military Medical University (Naval Medical University), Shanghai 200433, China; Corresponding authors.Center for Basic Research and Innovation of Medicine and Pharmacy (MOE), School of Pharmacy, Second Military Medical University (Naval Medical University), Shanghai 200433, China; Corresponding authors.Membrane protein degradation is a cutting-edge field in targeted protein degradation (TPD). Herein, we developed glypican-3 (GPC3)-mediated lysosome-targeting chimeras (GLTACs) as a novel strategy for the targeted degradation of tumor-specific membrane proteins. GLTACs utilize tumor-specific expression and endocytosis properties of GPC3 to degrade membrane proteins. By conjugating a GPC3-targeting peptide with the ligand of protein of interest (POI), GLTACs induce the formation of a ternary complex that is internalized into lysosomes, leading to the degradation of the POI. The effectiveness and specificity of GLTACs were validated by designing PD-L1, c-Met, and FGFR1 degraders. In particular, GLTAC WP0 potently degraded PD-L1 and induced T-cell-mediated tumor killing against HepG2 cells, highlighting the potential therapeutic applications. The development of GLTAC technology expands the scope of TPD strategies and opens new avenues for discovering novel therapeutic modalities against challenging protein targets.http://www.sciencedirect.com/science/article/pii/S2211383525001339Glypican-3PD-L1Lysosomal degradationMembrane proteinTargeted protein degradation
spellingShingle Yuxin Fang
Yaojin Zhu
Wei Wang
Zhewei Xia
Shipeng He
Guoqiang Dong
Chunquan Sheng
GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins
Acta Pharmaceutica Sinica B
Glypican-3
PD-L1
Lysosomal degradation
Membrane protein
Targeted protein degradation
title GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins
title_full GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins
title_fullStr GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins
title_full_unstemmed GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins
title_short GPC3-mediated lysosome-targeting chimeras (GLTACs) for targeted degradation of membrane proteins
title_sort gpc3 mediated lysosome targeting chimeras gltacs for targeted degradation of membrane proteins
topic Glypican-3
PD-L1
Lysosomal degradation
Membrane protein
Targeted protein degradation
url http://www.sciencedirect.com/science/article/pii/S2211383525001339
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