DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5

Abstract Spinal and bulbar muscular atrophy (SBMA) is a polyglutamine‐mediated neuromuscular disease caused by a CAG repeat expansion in the androgen receptor (AR) gene. While transcriptional dysregulation is known to play a critical role in the pathogenesis of SBMA, the underlying molecular pathome...

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Main Authors: Naohide Kondo, Genki Tohnai, Kentaro Sahashi, Madoka Iida, Mayumi Kataoka, Hideaki Nakatsuji, Yutaka Tsutsumi, Atsushi Hashizume, Hiroaki Adachi, Haruki Koike, Keiko Shinjo, Yutaka Kondo, Gen Sobue, Masahisa Katsuno
Format: Article
Language:English
Published: Springer Nature 2019-04-01
Series:EMBO Molecular Medicine
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Online Access:https://doi.org/10.15252/emmm.201708547
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author Naohide Kondo
Genki Tohnai
Kentaro Sahashi
Madoka Iida
Mayumi Kataoka
Hideaki Nakatsuji
Yutaka Tsutsumi
Atsushi Hashizume
Hiroaki Adachi
Haruki Koike
Keiko Shinjo
Yutaka Kondo
Gen Sobue
Masahisa Katsuno
author_facet Naohide Kondo
Genki Tohnai
Kentaro Sahashi
Madoka Iida
Mayumi Kataoka
Hideaki Nakatsuji
Yutaka Tsutsumi
Atsushi Hashizume
Hiroaki Adachi
Haruki Koike
Keiko Shinjo
Yutaka Kondo
Gen Sobue
Masahisa Katsuno
author_sort Naohide Kondo
collection DOAJ
description Abstract Spinal and bulbar muscular atrophy (SBMA) is a polyglutamine‐mediated neuromuscular disease caused by a CAG repeat expansion in the androgen receptor (AR) gene. While transcriptional dysregulation is known to play a critical role in the pathogenesis of SBMA, the underlying molecular pathomechanisms remain unclear. DNA methylation is a fundamental epigenetic modification that silences the transcription of various genes that have a CpG‐rich promoter. Here, we showed that DNA methyltransferase 1 (Dnmt1) is highly expressed in the spinal motor neurons of an SBMA mouse model and in patients with SBMA. Both genetic Dnmt1 depletion and treatment with RG108, a DNA methylation inhibitor, ameliorated the viability of SBMA model cells. Furthermore, a continuous intracerebroventricular injection of RG108 mitigated the phenotype of SBMA mice. DNA methylation array analysis identified hairy and enhancer of split 5 (Hes5) as having a CpG island with hyper‐methylation in the promoter region, and the Hes5 expression was strongly silenced in SBMA. Moreover, Hes5 over‐expression rescued the SBMA cells possibly by inducing Smad2 phosphorylation. Our findings suggest DNA hyper‐methylation underlies the neurodegeneration in SBMA.
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spelling doaj-art-3b53acade81c4e8ebab35318515fcdcb2025-08-20T02:11:22ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842019-04-0111511910.15252/emmm.201708547DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5Naohide Kondo0Genki Tohnai1Kentaro Sahashi2Madoka Iida3Mayumi Kataoka4Hideaki Nakatsuji5Yutaka Tsutsumi6Atsushi Hashizume7Hiroaki Adachi8Haruki Koike9Keiko Shinjo10Yutaka Kondo11Gen Sobue12Masahisa Katsuno13Department of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDepartment of Neurology, University of Occupational and Environmental Health School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineDivision of Cancer Biology, Nagoya University Graduate School of MedicineDivision of Cancer Biology, Nagoya University Graduate School of MedicineResearch Division of Dementia and Neurodegenerative Disease, Nagoya University Graduate School of MedicineDepartment of Neurology, Nagoya University Graduate School of MedicineAbstract Spinal and bulbar muscular atrophy (SBMA) is a polyglutamine‐mediated neuromuscular disease caused by a CAG repeat expansion in the androgen receptor (AR) gene. While transcriptional dysregulation is known to play a critical role in the pathogenesis of SBMA, the underlying molecular pathomechanisms remain unclear. DNA methylation is a fundamental epigenetic modification that silences the transcription of various genes that have a CpG‐rich promoter. Here, we showed that DNA methyltransferase 1 (Dnmt1) is highly expressed in the spinal motor neurons of an SBMA mouse model and in patients with SBMA. Both genetic Dnmt1 depletion and treatment with RG108, a DNA methylation inhibitor, ameliorated the viability of SBMA model cells. Furthermore, a continuous intracerebroventricular injection of RG108 mitigated the phenotype of SBMA mice. DNA methylation array analysis identified hairy and enhancer of split 5 (Hes5) as having a CpG island with hyper‐methylation in the promoter region, and the Hes5 expression was strongly silenced in SBMA. Moreover, Hes5 over‐expression rescued the SBMA cells possibly by inducing Smad2 phosphorylation. Our findings suggest DNA hyper‐methylation underlies the neurodegeneration in SBMA.https://doi.org/10.15252/emmm.201708547DNA methylationepigeneticsHes5RG108spinal and bulbar muscular atrophy
spellingShingle Naohide Kondo
Genki Tohnai
Kentaro Sahashi
Madoka Iida
Mayumi Kataoka
Hideaki Nakatsuji
Yutaka Tsutsumi
Atsushi Hashizume
Hiroaki Adachi
Haruki Koike
Keiko Shinjo
Yutaka Kondo
Gen Sobue
Masahisa Katsuno
DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5
EMBO Molecular Medicine
DNA methylation
epigenetics
Hes5
RG108
spinal and bulbar muscular atrophy
title DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5
title_full DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5
title_fullStr DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5
title_full_unstemmed DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5
title_short DNA methylation inhibitor attenuates polyglutamine‐induced neurodegeneration by regulating Hes5
title_sort dna methylation inhibitor attenuates polyglutamine induced neurodegeneration by regulating hes5
topic DNA methylation
epigenetics
Hes5
RG108
spinal and bulbar muscular atrophy
url https://doi.org/10.15252/emmm.201708547
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