Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease

Abstract Aberrant localization of proteins to mitochondria disturbs mitochondrial function and contributes to the pathogenesis of Huntington’s disease (HD). However, the crucial factors and the molecular mechanisms remain elusive. Here, we found that heat shock transcription factor 1 (HSF1) accumula...

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Main Authors: Chunyue Liu, Zixing Fu, Shanshan Wu, Xiaosong Wang, Shengrong Zhang, Chu Chu, Yuan Hong, Wenbo Wu, Shengqi Chen, Yueqing Jiang, Yang Wu, Yongbo Song, Yan Liu, Xing Guo
Format: Article
Language:English
Published: Springer Nature 2022-06-01
Series:EMBO Molecular Medicine
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Online Access:https://doi.org/10.15252/emmm.202215851
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author Chunyue Liu
Zixing Fu
Shanshan Wu
Xiaosong Wang
Shengrong Zhang
Chu Chu
Yuan Hong
Wenbo Wu
Shengqi Chen
Yueqing Jiang
Yang Wu
Yongbo Song
Yan Liu
Xing Guo
author_facet Chunyue Liu
Zixing Fu
Shanshan Wu
Xiaosong Wang
Shengrong Zhang
Chu Chu
Yuan Hong
Wenbo Wu
Shengqi Chen
Yueqing Jiang
Yang Wu
Yongbo Song
Yan Liu
Xing Guo
author_sort Chunyue Liu
collection DOAJ
description Abstract Aberrant localization of proteins to mitochondria disturbs mitochondrial function and contributes to the pathogenesis of Huntington’s disease (HD). However, the crucial factors and the molecular mechanisms remain elusive. Here, we found that heat shock transcription factor 1 (HSF1) accumulates in the mitochondria of HD cell models, a YAC128 mouse model, and human striatal organoids derived from HD induced pluripotent stem cells (iPSCs). Overexpression of mitochondria‐targeting HSF1 (mtHSF1) in the striatum causes neurodegeneration and HD‐like behavior in mice. Mechanistically, mtHSF1 facilitates mitochondrial fission by activating dynamin‐related protein 1 (Drp1) phosphorylation at S616. Moreover, mtHSF1 suppresses single‐stranded DNA‐binding protein 1 (SSBP1) oligomer formation, which results in mitochondrial DNA (mtDNA) deletion. The suppression of HSF1 mitochondrial localization by DH1, a unique peptide inhibitor, abolishes HSF1‐induced mitochondrial abnormalities and ameliorates deficits in an HD animal model and human striatal organoids. Altogether, our findings describe an unsuspected role of HSF1 in contributing to mitochondrial dysfunction, which may provide a promising therapeutic target for HD.
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spelling doaj-art-5ccc4c519e7e441a9304214eb407cf672025-08-20T03:06:00ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842022-06-0114711910.15252/emmm.202215851Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's diseaseChunyue Liu0Zixing Fu1Shanshan Wu2Xiaosong Wang3Shengrong Zhang4Chu Chu5Yuan Hong6Wenbo Wu7Shengqi Chen8Yueqing Jiang9Yang Wu10Yongbo Song11Yan Liu12Xing Guo13State Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Interdisciplinary InnoCenter for Organoids, Institute for Stem Cell and Neural Regeneration, School of Pharmacy, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Interdisciplinary InnoCenter for Organoids, Institute for Stem Cell and Neural Regeneration, School of Pharmacy, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Interdisciplinary InnoCenter for Organoids, Institute for Stem Cell and Neural Regeneration, School of Pharmacy, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityState Key Laboratory of Magnetic Resonance and Atomic and Molecular Physics, Key Laboratory of Magnetic Resonance in Biological Systems, Wuhan Center for Magnetic Resonance, Innovation Academy for Precision Measurement Science and Technology, Chinese Academy of SciencesDepartment of Pharmacology, Shenyang Pharmaceutical UniversityState Key Laboratory of Reproductive Medicine, Interdisciplinary InnoCenter for Organoids, Institute for Stem Cell and Neural Regeneration, School of Pharmacy, Nanjing Medical UniversityState Key Laboratory of Reproductive Medicine, Key Laboratory of Human Functional Genomics of Jiangsu Province, Department of Neurobiology, Interdisciplinary InnoCenter for Organoids, School of Basic Medical Sciences, Nanjing Medical UniversityAbstract Aberrant localization of proteins to mitochondria disturbs mitochondrial function and contributes to the pathogenesis of Huntington’s disease (HD). However, the crucial factors and the molecular mechanisms remain elusive. Here, we found that heat shock transcription factor 1 (HSF1) accumulates in the mitochondria of HD cell models, a YAC128 mouse model, and human striatal organoids derived from HD induced pluripotent stem cells (iPSCs). Overexpression of mitochondria‐targeting HSF1 (mtHSF1) in the striatum causes neurodegeneration and HD‐like behavior in mice. Mechanistically, mtHSF1 facilitates mitochondrial fission by activating dynamin‐related protein 1 (Drp1) phosphorylation at S616. Moreover, mtHSF1 suppresses single‐stranded DNA‐binding protein 1 (SSBP1) oligomer formation, which results in mitochondrial DNA (mtDNA) deletion. The suppression of HSF1 mitochondrial localization by DH1, a unique peptide inhibitor, abolishes HSF1‐induced mitochondrial abnormalities and ameliorates deficits in an HD animal model and human striatal organoids. Altogether, our findings describe an unsuspected role of HSF1 in contributing to mitochondrial dysfunction, which may provide a promising therapeutic target for HD.https://doi.org/10.15252/emmm.202215851heat shock transcription factor 1human striatal organoidsHuntington's diseasemitochondrial DNAsingle‐stranded DNA‐binding protein 1
spellingShingle Chunyue Liu
Zixing Fu
Shanshan Wu
Xiaosong Wang
Shengrong Zhang
Chu Chu
Yuan Hong
Wenbo Wu
Shengqi Chen
Yueqing Jiang
Yang Wu
Yongbo Song
Yan Liu
Xing Guo
Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease
EMBO Molecular Medicine
heat shock transcription factor 1
human striatal organoids
Huntington's disease
mitochondrial DNA
single‐stranded DNA‐binding protein 1
title Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease
title_full Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease
title_fullStr Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease
title_full_unstemmed Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease
title_short Mitochondrial HSF1 triggers mitochondrial dysfunction and neurodegeneration in Huntington's disease
title_sort mitochondrial hsf1 triggers mitochondrial dysfunction and neurodegeneration in huntington s disease
topic heat shock transcription factor 1
human striatal organoids
Huntington's disease
mitochondrial DNA
single‐stranded DNA‐binding protein 1
url https://doi.org/10.15252/emmm.202215851
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