A humanized Gs-coupled DREADD for circuit and behavior modulation

Designer receptors exclusively activated by designer drugs (DREADDs) play important roles in neuroscience research and show great promise for future clinical interventions in neurological diseases. The Gs-coupled DREADD, rM3Ds, modulates excitability in neuron subsets that are sensitive to downstrea...

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Main Authors: Qi Zhang, Ruiqi Wang, Liang Zhang, Mengqi Li, Jianbang Lin, Xiaoyang Lu, Yixuan Tian, Yunping Lin, Taian Liu, Yefei Chen, Yuantao Li, Jun Cao, Qiang Wu, Jinhui Wang, Zhonghua Lu, Zexuan Hong
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-04-01
Series:Frontiers in Cellular Neuroscience
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Online Access:https://www.frontiersin.org/articles/10.3389/fncel.2025.1577117/full
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author Qi Zhang
Ruiqi Wang
Liang Zhang
Mengqi Li
Jianbang Lin
Jianbang Lin
Jianbang Lin
Xiaoyang Lu
Yixuan Tian
Yunping Lin
Yunping Lin
Yunping Lin
Taian Liu
Yefei Chen
Yuantao Li
Jun Cao
Qiang Wu
Qiang Wu
Jinhui Wang
Zhonghua Lu
Zhonghua Lu
Zhonghua Lu
Zhonghua Lu
Zexuan Hong
author_facet Qi Zhang
Ruiqi Wang
Liang Zhang
Mengqi Li
Jianbang Lin
Jianbang Lin
Jianbang Lin
Xiaoyang Lu
Yixuan Tian
Yunping Lin
Yunping Lin
Yunping Lin
Taian Liu
Yefei Chen
Yuantao Li
Jun Cao
Qiang Wu
Qiang Wu
Jinhui Wang
Zhonghua Lu
Zhonghua Lu
Zhonghua Lu
Zhonghua Lu
Zexuan Hong
author_sort Qi Zhang
collection DOAJ
description Designer receptors exclusively activated by designer drugs (DREADDs) play important roles in neuroscience research and show great promise for future clinical interventions in neurological diseases. The Gs-coupled DREADD, rM3Ds, modulates excitability in neuron subsets that are sensitive to downstream effectors of Gs protein. However, given the non-human nature of the rM3Ds backbone, risks about potential immunogenicity and tolerability exist when considering clinical translation. Here, we report the development of a whole sequence-humanized Gs-coupled DREADD, hM3Ds. We found that hM3Ds has a comparable DREADD ligand response profile to rM3Ds. We then selectively expressed hM3Ds in D1 medium spiny neurons (D1-MSNs) and found that hM3Ds was able to activate the D1-MSNs-mediated basal ganglia direct pathway and alleviate Parkinsonian phenotypes in a Parkinson’s disease mouse model. In conclusion, this engineered humanized Gs-coupled DREADD is suitable as an effective, and likely safer, DREADD tool for both research and future clinical applications.
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spelling doaj-art-c5a4df6f17c34cff92eb4717fabe44da2025-08-20T03:17:27ZengFrontiers Media S.A.Frontiers in Cellular Neuroscience1662-51022025-04-011910.3389/fncel.2025.15771171577117A humanized Gs-coupled DREADD for circuit and behavior modulationQi Zhang0Ruiqi Wang1Liang Zhang2Mengqi Li3Jianbang Lin4Jianbang Lin5Jianbang Lin6Xiaoyang Lu7Yixuan Tian8Yunping Lin9Yunping Lin10Yunping Lin11Taian Liu12Yefei Chen13Yuantao Li14Jun Cao15Qiang Wu16Qiang Wu17Jinhui Wang18Zhonghua Lu19Zhonghua Lu20Zhonghua Lu21Zhonghua Lu22Zexuan Hong23Department of Medicinal Chemistry and Natural Medicine Chemistry, College of Pharmacy, Harbin Medical University, Harbin, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaDepartment of Anesthesiology, The Third People’s Hospital of Shenzhen, Shenzhen, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaShenzhen Key Laboratory for Molecular Biology of Neural Development, Shenzhen Technological Research Center for Primate Translational Medicine, Shenzhen-Hong Kong Institute of Brain Science, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaUniversity of Chinese Academy of Sciences, Beijing, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaShenzhen Key Laboratory for Molecular Biology of Neural Development, Shenzhen Technological Research Center for Primate Translational Medicine, Shenzhen-Hong Kong Institute of Brain Science, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaUniversity of Chinese Academy of Sciences, Beijing, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaShenzhen Maternity and Child Healthcare Hospital, Southern Medical University, Shenzhen, ChinaShenzhen Maternity and Child Healthcare Hospital, Southern Medical University, Shenzhen, ChinaDepartment of Anesthesiology, The Third People’s Hospital of Shenzhen, Shenzhen, ChinaState Key Laboratory of Biomedical Imaging Science and System, Key Laboratory of Biomedical Imaging Science and System, Chinese Academy of Sciences, Shenzhen, ChinaDepartment of Medicinal Chemistry and Natural Medicine Chemistry, College of Pharmacy, Harbin Medical University, Harbin, ChinaResearch Center for Primate Neuromodulation and Neuroimaging, Institute of Biomedical and Health Engineering, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaShenzhen Key Laboratory for Molecular Biology of Neural Development, Shenzhen Technological Research Center for Primate Translational Medicine, Shenzhen-Hong Kong Institute of Brain Science, Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen, ChinaUniversity of Chinese Academy of Sciences, Beijing, ChinaState Key Laboratory of Biomedical Imaging Science and System, Key Laboratory of Biomedical Imaging Science and System, Chinese Academy of Sciences, Shenzhen, ChinaShenzhen Maternity and Child Healthcare Hospital, Southern Medical University, Shenzhen, ChinaDesigner receptors exclusively activated by designer drugs (DREADDs) play important roles in neuroscience research and show great promise for future clinical interventions in neurological diseases. The Gs-coupled DREADD, rM3Ds, modulates excitability in neuron subsets that are sensitive to downstream effectors of Gs protein. However, given the non-human nature of the rM3Ds backbone, risks about potential immunogenicity and tolerability exist when considering clinical translation. Here, we report the development of a whole sequence-humanized Gs-coupled DREADD, hM3Ds. We found that hM3Ds has a comparable DREADD ligand response profile to rM3Ds. We then selectively expressed hM3Ds in D1 medium spiny neurons (D1-MSNs) and found that hM3Ds was able to activate the D1-MSNs-mediated basal ganglia direct pathway and alleviate Parkinsonian phenotypes in a Parkinson’s disease mouse model. In conclusion, this engineered humanized Gs-coupled DREADD is suitable as an effective, and likely safer, DREADD tool for both research and future clinical applications.https://www.frontiersin.org/articles/10.3389/fncel.2025.1577117/fullDREADDneuronal activationmodulationtransgene modificationGs-signalingD1-MSNs
spellingShingle Qi Zhang
Ruiqi Wang
Liang Zhang
Mengqi Li
Jianbang Lin
Jianbang Lin
Jianbang Lin
Xiaoyang Lu
Yixuan Tian
Yunping Lin
Yunping Lin
Yunping Lin
Taian Liu
Yefei Chen
Yuantao Li
Jun Cao
Qiang Wu
Qiang Wu
Jinhui Wang
Zhonghua Lu
Zhonghua Lu
Zhonghua Lu
Zhonghua Lu
Zexuan Hong
A humanized Gs-coupled DREADD for circuit and behavior modulation
Frontiers in Cellular Neuroscience
DREADD
neuronal activation
modulation
transgene modification
Gs-signaling
D1-MSNs
title A humanized Gs-coupled DREADD for circuit and behavior modulation
title_full A humanized Gs-coupled DREADD for circuit and behavior modulation
title_fullStr A humanized Gs-coupled DREADD for circuit and behavior modulation
title_full_unstemmed A humanized Gs-coupled DREADD for circuit and behavior modulation
title_short A humanized Gs-coupled DREADD for circuit and behavior modulation
title_sort humanized gs coupled dreadd for circuit and behavior modulation
topic DREADD
neuronal activation
modulation
transgene modification
Gs-signaling
D1-MSNs
url https://www.frontiersin.org/articles/10.3389/fncel.2025.1577117/full
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