Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis

Hereditary transthyretin (TTR) amyloidosis (ATTRv amyloidosis) is an autosomal dominant disease caused by various TTR mutations. Despite the fact that ATTR Tyr114Cys (p.Tyr134Cys) amyloidosis (tyrosine to cysteine at codon 114) exhibits poorer prognosis than other ATTRv amyloidosis and leads to deat...

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Main Authors: Kenta Ouchi, Takeshi Masuda, Kou Yonemaru, Kaori Isono, Yuki Ohya, Nobuaki Shiraki, Masayoshi Tasaki, Yukihiro Inomata, Mitsuharu Ueda, Takumi Era, Shoen Kume, Yukio Ando, Hirofumi Jono
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Language:English
Published: Elsevier 2025-06-01
Series:Biochemistry and Biophysics Reports
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Online Access:http://www.sciencedirect.com/science/article/pii/S2405580825000998
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author Kenta Ouchi
Takeshi Masuda
Kou Yonemaru
Kaori Isono
Yuki Ohya
Nobuaki Shiraki
Masayoshi Tasaki
Yukihiro Inomata
Mitsuharu Ueda
Takumi Era
Shoen Kume
Yukio Ando
Hirofumi Jono
author_facet Kenta Ouchi
Takeshi Masuda
Kou Yonemaru
Kaori Isono
Yuki Ohya
Nobuaki Shiraki
Masayoshi Tasaki
Yukihiro Inomata
Mitsuharu Ueda
Takumi Era
Shoen Kume
Yukio Ando
Hirofumi Jono
author_sort Kenta Ouchi
collection DOAJ
description Hereditary transthyretin (TTR) amyloidosis (ATTRv amyloidosis) is an autosomal dominant disease caused by various TTR mutations. Despite the fact that ATTR Tyr114Cys (p.Tyr134Cys) amyloidosis (tyrosine to cysteine at codon 114) exhibits poorer prognosis than other ATTRv amyloidosis and leads to death due to severe clinical symptoms, the molecular pathogenesis of ATTR Tyr114Cys amyloidosis is still largely unknown. In this study, we took advantage of ATTR Tyr114Cys amyloidosis-specific induced pluripotent stem (iPS) cells to differentiate into hepatocyte-like cells (Y114C-HLCs), which are mainly TTR producing cells, and elucidated their pathogenesis. We performed proteomic analysis to comprehensively identify specific intracellular signaling pathways involved in Y114C-HLCs, and identified the specific proteins changed only in Y114C-HLCs, in comparison with disease control HLCs from ATTR Val30Met amyloidosis (V30M-HLCs). Moreover, we have succeeded in identifying several specific intracellular signals that are significantly activated in Y114C-HLCs, including cellular responses to stress and extracellular matrix organization. Our proteomic analysis is the first to report that the specific point mutations in ATTRv amyloidosis cause dynamic changes in cellular response, and reveal the specific intracellular signals may be involved in the specific pathogenesis of ATTR Tyr114Cys amyloidosis.
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spelling doaj-art-3f9b2bdb647e42aa9bf668eb2c19b2832025-08-20T02:17:09ZengElsevierBiochemistry and Biophysics Reports2405-58082025-06-014210201210.1016/j.bbrep.2025.102012Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosisKenta Ouchi0Takeshi Masuda1Kou Yonemaru2Kaori Isono3Yuki Ohya4Nobuaki Shiraki5Masayoshi Tasaki6Yukihiro Inomata7Mitsuharu Ueda8Takumi Era9Shoen Kume10Yukio Ando11Hirofumi Jono12Department of Clinical Pharmaceutical Sciences, Graduate School of Pharmaceutical Sciences, Kumamoto University, 2-2-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, JapanGraduate School of Media and Governance / Institute for Advanced Biosciences, Keio University, 14-1 Baba-machi, Tsuruoka-City, Yamagata Prefecture, 997-0035, JapanDepartment of Clinical Pharmaceutical Sciences, Graduate School of Pharmaceutical Sciences, Kumamoto University, 2-2-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, JapanDepartment of Transplantation and Paediatric Surgery, Graduate School of Medical Science, Kumamoto University, 1-1-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, JapanDepartment of Transplantation and Paediatric Surgery, Graduate School of Medical Science, Kumamoto University, 1-1-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, Japan; Department of Pediatric Surgery, Kumamoto Rosai Hospital, 1670 Takehara-cho, Yatsushiro City, Kumamoto Prefecture, 866-0826, JapanSchool of Life Science and Technology, Tokyo Institute of Technology, 4259 Nagatsuta-cho, Midori Ward, Yokohama City, Kanagawa Prefecture, 226-8501, JapanDepartment of Biomedical Laboratory Sciences, Graduate School of Health Sciences, Kumamoto University, Kumamoto, 1-1-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, Japan; Department of Neurology, Graduate School of Medical Science, Kumamoto University, 1-1-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, JapanDepartment of Pediatric Surgery, Kumamoto Rosai Hospital, 1670 Takehara-cho, Yatsushiro City, Kumamoto Prefecture, 866-0826, JapanDepartment of Neurology, Graduate School of Medical Science, Kumamoto University, 1-1-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, JapanDepartment of Cell Modulation, Institute of Molecular Embryology and Genetics, Kumamoto University, 2-2-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, JapanSchool of Life Science and Technology, Tokyo Institute of Technology, 4259 Nagatsuta-cho, Midori Ward, Yokohama City, Kanagawa Prefecture, 226-8501, JapanDepartment of Amyloidosis Research, Nagasaki International University, 2825-7 Huis Ten Bosch Cho, Sasebo City, Nagasaki Prefecture, 859-3298, JapanDepartment of Clinical Pharmaceutical Sciences, Graduate School of Pharmaceutical Sciences, Kumamoto University, 2-2-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, Japan; Department of Pharmacy, Kumamoto University Hospital, 1-1-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, Japan; Corresponding author. Department of Clinical Pharmaceutical Sciences, Graduate School of Pharmaceutical Sciences, Kumamoto University, 2-2-1 Honjo, Chuo Ward, Kumamoto City, Kumamoto Prefecture, 860-8556, Japan.Hereditary transthyretin (TTR) amyloidosis (ATTRv amyloidosis) is an autosomal dominant disease caused by various TTR mutations. Despite the fact that ATTR Tyr114Cys (p.Tyr134Cys) amyloidosis (tyrosine to cysteine at codon 114) exhibits poorer prognosis than other ATTRv amyloidosis and leads to death due to severe clinical symptoms, the molecular pathogenesis of ATTR Tyr114Cys amyloidosis is still largely unknown. In this study, we took advantage of ATTR Tyr114Cys amyloidosis-specific induced pluripotent stem (iPS) cells to differentiate into hepatocyte-like cells (Y114C-HLCs), which are mainly TTR producing cells, and elucidated their pathogenesis. We performed proteomic analysis to comprehensively identify specific intracellular signaling pathways involved in Y114C-HLCs, and identified the specific proteins changed only in Y114C-HLCs, in comparison with disease control HLCs from ATTR Val30Met amyloidosis (V30M-HLCs). Moreover, we have succeeded in identifying several specific intracellular signals that are significantly activated in Y114C-HLCs, including cellular responses to stress and extracellular matrix organization. Our proteomic analysis is the first to report that the specific point mutations in ATTRv amyloidosis cause dynamic changes in cellular response, and reveal the specific intracellular signals may be involved in the specific pathogenesis of ATTR Tyr114Cys amyloidosis.http://www.sciencedirect.com/science/article/pii/S2405580825000998ATTR amyloidosisTransthyretinInduced pluripotent stem cellsProteomic analysis
spellingShingle Kenta Ouchi
Takeshi Masuda
Kou Yonemaru
Kaori Isono
Yuki Ohya
Nobuaki Shiraki
Masayoshi Tasaki
Yukihiro Inomata
Mitsuharu Ueda
Takumi Era
Shoen Kume
Yukio Ando
Hirofumi Jono
Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis
Biochemistry and Biophysics Reports
ATTR amyloidosis
Transthyretin
Induced pluripotent stem cells
Proteomic analysis
title Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis
title_full Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis
title_fullStr Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis
title_full_unstemmed Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis
title_short Dynamic changes of intracellular signals in ATTR Tyr114Cys amyloidosis
title_sort dynamic changes of intracellular signals in attr tyr114cys amyloidosis
topic ATTR amyloidosis
Transthyretin
Induced pluripotent stem cells
Proteomic analysis
url http://www.sciencedirect.com/science/article/pii/S2405580825000998
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