c-Myc-Targeting PROTAC Based on a TNA-DNA Bivalent Binder for Combination Therapy of Triple-Negative Breast Cancer

Triple-negative breast cancer (TNBC) is highly aggressive with a poor clinical prognosis and no targeted therapy. The c-Myc protein is a master transcription factor and a potential therapeutic target for TNBC. In this study, we develop a PROTAC (PROteolysis TArgeting Chimera) based on TNA (threose n...

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Veröffentlicht in:Journal of the American Chemical Society Jg. 145; H. 16; S. 9334
Hauptverfasser: Li, Xintong, Zhang, Ze, Gao, Fangyan, Ma, Yuxuan, Wei, Dongying, Lu, Zhangwei, Chen, Siqi, Wang, Mengqi, Wang, Yueyao, Xu, Kun, Wang, Runtian, Xu, Feng, Chen, Jia-Yu, Zhu, Chengjun, Li, Zhe, Yu, Hanyang, Guan, Xiaoxiang
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Sprache:Englisch
Veröffentlicht: United States 26.04.2023
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ISSN:1520-5126, 1520-5126
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Abstract Triple-negative breast cancer (TNBC) is highly aggressive with a poor clinical prognosis and no targeted therapy. The c-Myc protein is a master transcription factor and a potential therapeutic target for TNBC. In this study, we develop a PROTAC (PROteolysis TArgeting Chimera) based on TNA (threose nucleic acid) and DNA that effectively targets and degrades c-Myc. The TNA aptamer is selected in vitro to bind the c-Myc/Max heterodimer and appended to the E-box DNA sequence to create a high-affinity, biologically stable bivalent binder. The TNA-E box-pomalidomide (TEP) conjugate specifically degrades endogenous c-Myc/Max, inhibits TNBC cell proliferation, and sensitizes TNBC cells to the cyclin-dependent kinase inhibitor palbociclib in vitro. In a mouse TNBC model, combination therapy with TEP and palbociclib potently suppresses tumor growth. This study offers a promising nucleic acid-based PROTAC modality for both chemical biology studies and therapeutic interventions of TNBC.
AbstractList Triple-negative breast cancer (TNBC) is highly aggressive with a poor clinical prognosis and no targeted therapy. The c-Myc protein is a master transcription factor and a potential therapeutic target for TNBC. In this study, we develop a PROTAC (PROteolysis TArgeting Chimera) based on TNA (threose nucleic acid) and DNA that effectively targets and degrades c-Myc. The TNA aptamer is selected in vitro to bind the c-Myc/Max heterodimer and appended to the E-box DNA sequence to create a high-affinity, biologically stable bivalent binder. The TNA-E box-pomalidomide (TEP) conjugate specifically degrades endogenous c-Myc/Max, inhibits TNBC cell proliferation, and sensitizes TNBC cells to the cyclin-dependent kinase inhibitor palbociclib in vitro. In a mouse TNBC model, combination therapy with TEP and palbociclib potently suppresses tumor growth. This study offers a promising nucleic acid-based PROTAC modality for both chemical biology studies and therapeutic interventions of TNBC.Triple-negative breast cancer (TNBC) is highly aggressive with a poor clinical prognosis and no targeted therapy. The c-Myc protein is a master transcription factor and a potential therapeutic target for TNBC. In this study, we develop a PROTAC (PROteolysis TArgeting Chimera) based on TNA (threose nucleic acid) and DNA that effectively targets and degrades c-Myc. The TNA aptamer is selected in vitro to bind the c-Myc/Max heterodimer and appended to the E-box DNA sequence to create a high-affinity, biologically stable bivalent binder. The TNA-E box-pomalidomide (TEP) conjugate specifically degrades endogenous c-Myc/Max, inhibits TNBC cell proliferation, and sensitizes TNBC cells to the cyclin-dependent kinase inhibitor palbociclib in vitro. In a mouse TNBC model, combination therapy with TEP and palbociclib potently suppresses tumor growth. This study offers a promising nucleic acid-based PROTAC modality for both chemical biology studies and therapeutic interventions of TNBC.
Triple-negative breast cancer (TNBC) is highly aggressive with a poor clinical prognosis and no targeted therapy. The c-Myc protein is a master transcription factor and a potential therapeutic target for TNBC. In this study, we develop a PROTAC (PROteolysis TArgeting Chimera) based on TNA (threose nucleic acid) and DNA that effectively targets and degrades c-Myc. The TNA aptamer is selected in vitro to bind the c-Myc/Max heterodimer and appended to the E-box DNA sequence to create a high-affinity, biologically stable bivalent binder. The TNA-E box-pomalidomide (TEP) conjugate specifically degrades endogenous c-Myc/Max, inhibits TNBC cell proliferation, and sensitizes TNBC cells to the cyclin-dependent kinase inhibitor palbociclib in vitro. In a mouse TNBC model, combination therapy with TEP and palbociclib potently suppresses tumor growth. This study offers a promising nucleic acid-based PROTAC modality for both chemical biology studies and therapeutic interventions of TNBC.
Author Wang, Mengqi
Li, Xintong
Li, Zhe
Ma, Yuxuan
Wei, Dongying
Lu, Zhangwei
Wang, Yueyao
Wang, Runtian
Yu, Hanyang
Gao, Fangyan
Zhang, Ze
Xu, Feng
Xu, Kun
Chen, Jia-Yu
Zhu, Chengjun
Guan, Xiaoxiang
Chen, Siqi
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  surname: Li
  fullname: Li, Xintong
  organization: Department of Oncology, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210029, China
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  givenname: Ze
  orcidid: 0000-0002-8570-8790
  surname: Zhang
  fullname: Zhang, Ze
  organization: State Key Laboratory of Coordination Chemistry, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Jiangsu Key Laboratory of Artificial Functional Materials, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
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  givenname: Fangyan
  surname: Gao
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  organization: Department of Oncology, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210029, China
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  givenname: Yuxuan
  surname: Ma
  fullname: Ma, Yuxuan
  organization: State Key Laboratory of Analytical Chemistry for Life Science, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Nanjing University, Nanjing 210023, China
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  givenname: Dongying
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  fullname: Wei, Dongying
  organization: State Key Laboratory of Coordination Chemistry, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Jiangsu Key Laboratory of Artificial Functional Materials, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
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  givenname: Zhangwei
  surname: Lu
  fullname: Lu, Zhangwei
  organization: State Key Laboratory of Analytical Chemistry for Life Science, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Nanjing University, Nanjing 210023, China
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  givenname: Siqi
  surname: Chen
  fullname: Chen, Siqi
  organization: State Key Laboratory of Coordination Chemistry, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Jiangsu Key Laboratory of Artificial Functional Materials, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
– sequence: 8
  givenname: Mengqi
  surname: Wang
  fullname: Wang, Mengqi
  organization: State Key Laboratory of Pharmaceutical Biotechnology, School of Life Sciences, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
– sequence: 9
  givenname: Yueyao
  surname: Wang
  fullname: Wang, Yueyao
  organization: State Key Laboratory of Coordination Chemistry, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Jiangsu Key Laboratory of Artificial Functional Materials, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
– sequence: 10
  givenname: Kun
  surname: Xu
  fullname: Xu, Kun
  organization: Department of Oncology, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210029, China
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  orcidid: 0000-0001-7434-3944
  surname: Wang
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  organization: Department of Oncology, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210029, China
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  givenname: Feng
  surname: Xu
  fullname: Xu, Feng
  organization: Department of Oncology, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210029, China
– sequence: 13
  givenname: Jia-Yu
  surname: Chen
  fullname: Chen, Jia-Yu
  organization: State Key Laboratory of Pharmaceutical Biotechnology, School of Life Sciences, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
– sequence: 14
  givenname: Chengjun
  surname: Zhu
  fullname: Zhu, Chengjun
  organization: Department of Oncology, The First Affiliated Hospital of Nanjing Medical University, Nanjing 210029, China
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  givenname: Zhe
  orcidid: 0000-0002-2755-2409
  surname: Li
  fullname: Li, Zhe
  organization: State Key Laboratory of Analytical Chemistry for Life Science, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Nanjing University, Nanjing 210023, China
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  givenname: Hanyang
  orcidid: 0000-0003-1257-1735
  surname: Yu
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  organization: State Key Laboratory of Coordination Chemistry, Department of Biomedical Engineering, College of Engineering and Applied Sciences, Jiangsu Key Laboratory of Artificial Functional Materials, Chemistry and Biomedicine Innovation Center (ChemBIC), Nanjing University, Nanjing 210023, China
– sequence: 17
  givenname: Xiaoxiang
  surname: Guan
  fullname: Guan, Xiaoxiang
  organization: Jiangsu Key Lab of Cancer Biomarkers, Prevention and Treatment, Collaborative Innovation Center for Personalized Cancer Medicine, Nanjing Medical University, Nanjing 210029, China
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Snippet Triple-negative breast cancer (TNBC) is highly aggressive with a poor clinical prognosis and no targeted therapy. The c-Myc protein is a master transcription...
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SubjectTerms Animals
Antineoplastic Agents - pharmacology
Antineoplastic Agents - therapeutic use
Cell Line, Tumor
Cell Proliferation
Disease Models, Animal
Genes, myc
Humans
Mice
Transcription Factors
Triple Negative Breast Neoplasms - pathology
Title c-Myc-Targeting PROTAC Based on a TNA-DNA Bivalent Binder for Combination Therapy of Triple-Negative Breast Cancer
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