Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum

Hereditary transthyretin amyloidosis (ATTRv) results from genetic mutations that destabilize transthyretin (TTR), leading to the formation of extracellular aggregates and amyloid fibrils. A common pathological feature of ATTRv is the capacity of TTR variants to evade endoplasmic reticulum quality co...

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Main Authors: Jia Meng, Shan-jun Cai
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-07-01
Series:Frontiers in Physiology
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Online Access:https://www.frontiersin.org/articles/10.3389/fphys.2025.1623185/full
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author Jia Meng
Shan-jun Cai
Shan-jun Cai
Shan-jun Cai
Shan-jun Cai
author_facet Jia Meng
Shan-jun Cai
Shan-jun Cai
Shan-jun Cai
Shan-jun Cai
author_sort Jia Meng
collection DOAJ
description Hereditary transthyretin amyloidosis (ATTRv) results from genetic mutations that destabilize transthyretin (TTR), leading to the formation of extracellular aggregates and amyloid fibrils. A common pathological feature of ATTRv is the capacity of TTR variants to evade endoplasmic reticulum quality control (ERQC) and be secreted, underscoring the critical role of ER regulation in disease pathogenesis. Notably, the TTR Gly83Arg mutation causes familial vitreous amyloidosis, a subtype distinguished by abnormal TTR deposition in the ocular vitreous cavity. Current therapies for ATTRv are ineffective in crossing the blood-retinal barrier or in halting the progression of ocular amyloidosis. This review summarizes the molecular mechanisms of ER-regulated TTR secretion and explores potential causes of ocular amyloid deposition, aiming to provide mechanistic insights into familial vitreous amyloidosis.
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series Frontiers in Physiology
spelling doaj-art-548644edc8d8454fb7ea2d40eab4d8eb2025-08-20T03:15:54ZengFrontiers Media S.A.Frontiers in Physiology1664-042X2025-07-011610.3389/fphys.2025.16231851623185Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulumJia Meng0Shan-jun Cai1Shan-jun Cai2Shan-jun Cai3Shan-jun Cai4Department of Ophthalmology, Affiliated Hospital of Zunyi Medical University, Zunyi, ChinaDepartment of Ophthalmology, Affiliated Hospital of Zunyi Medical University, Zunyi, ChinaGuizhou Eye Hospital, Zunyi, ChinaGuizhou Branch of National Eye Disease Clinical Research Center, Zunyi, ChinaSpecial Key Laboratory of Ocular Diseases of Guizhou Province, Zunyi, ChinaHereditary transthyretin amyloidosis (ATTRv) results from genetic mutations that destabilize transthyretin (TTR), leading to the formation of extracellular aggregates and amyloid fibrils. A common pathological feature of ATTRv is the capacity of TTR variants to evade endoplasmic reticulum quality control (ERQC) and be secreted, underscoring the critical role of ER regulation in disease pathogenesis. Notably, the TTR Gly83Arg mutation causes familial vitreous amyloidosis, a subtype distinguished by abnormal TTR deposition in the ocular vitreous cavity. Current therapies for ATTRv are ineffective in crossing the blood-retinal barrier or in halting the progression of ocular amyloidosis. This review summarizes the molecular mechanisms of ER-regulated TTR secretion and explores potential causes of ocular amyloid deposition, aiming to provide mechanistic insights into familial vitreous amyloidosis.https://www.frontiersin.org/articles/10.3389/fphys.2025.1623185/fullendoplasmic reticulumendoplasmic reticulum quality controlunfolded protein responsetransthyretinvitreous amyloidosis
spellingShingle Jia Meng
Shan-jun Cai
Shan-jun Cai
Shan-jun Cai
Shan-jun Cai
Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum
Frontiers in Physiology
endoplasmic reticulum
endoplasmic reticulum quality control
unfolded protein response
transthyretin
vitreous amyloidosis
title Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum
title_full Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum
title_fullStr Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum
title_full_unstemmed Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum
title_short Secretion of transthyretin: molecular mechanisms dependent on the endoplasmic reticulum
title_sort secretion of transthyretin molecular mechanisms dependent on the endoplasmic reticulum
topic endoplasmic reticulum
endoplasmic reticulum quality control
unfolded protein response
transthyretin
vitreous amyloidosis
url https://www.frontiersin.org/articles/10.3389/fphys.2025.1623185/full
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AT shanjuncai secretionoftransthyretinmolecularmechanismsdependentontheendoplasmicreticulum
AT shanjuncai secretionoftransthyretinmolecularmechanismsdependentontheendoplasmicreticulum
AT shanjuncai secretionoftransthyretinmolecularmechanismsdependentontheendoplasmicreticulum
AT shanjuncai secretionoftransthyretinmolecularmechanismsdependentontheendoplasmicreticulum