The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy

Abstract Alzheimer's disease (AD) is a neurodegenerative disorder characterized by progressive cognitive decline, behavioral impairments, and psychiatric comorbidities. The pathogenesis of AD remains incompletely elucidated, despite advances in dominant hypotheses such as the β-amyloid (Aβ) cas...

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Main Authors: Xin Li, Ziyang Wu, Xiaying Si, Jing Li, Guode Wu, Manxia Wang
Format: Article
Language:English
Published: BMC 2025-05-01
Series:European Journal of Medical Research
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Online Access:https://doi.org/10.1186/s40001-025-02699-w
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author Xin Li
Ziyang Wu
Xiaying Si
Jing Li
Guode Wu
Manxia Wang
author_facet Xin Li
Ziyang Wu
Xiaying Si
Jing Li
Guode Wu
Manxia Wang
author_sort Xin Li
collection DOAJ
description Abstract Alzheimer's disease (AD) is a neurodegenerative disorder characterized by progressive cognitive decline, behavioral impairments, and psychiatric comorbidities. The pathogenesis of AD remains incompletely elucidated, despite advances in dominant hypotheses such as the β-amyloid (Aβ) cascade, tauopathy, cholinergic deficiency, and neuroinflammation mechanisms. However, these hypotheses inadequately explain the multifactorial nature of AD, which exposes limitations in our understanding of its mechanisms. Mitochondrial dysfunction is known to play a pivotal role in AD, and since patients exhibit intracellular mitochondrial dysfunction and structural changes in the brain at an early stage, correcting the imbalance of mitochondrial homeostasis and the cytopathological changes caused by it may be a potential target for early treatment of AD. Mitochondrial structural abnormalities accelerate AD pathogenesis. For instance, structural and functional alterations in the mitochondria-associated endoplasmic reticulum membrane (MAM) can disrupt intracellular Ca2⁺ homeostasis and cholesterol metabolism, consequently promoting Aβ accumulation. In addition, the overaccumulation of Aβ and hyperphosphorylated tau proteins can further damage neurons by disrupting mitochondrial integrity and mitophagy, thereby amplifying pathological aggregation and exacerbating neurodegeneration in AD. Furthermore, Aβ deposition and abnormal tau proteins can disrupt mitochondrial dynamics through dysregulation of fission/fusion proteins, leading to excessive mitochondrial fragmentation and subsequent dysfunction. Additionally, hyperphosphorylated tau proteins can impair mitochondrial transport, resulting in axonal dysfunction in AD. This article reviews the biological significance of mitochondrial structural morphology, dynamics, and mitochondrial DNA (mtDNA) instability in AD pathology, emphasizing mitophagy abnormalities as a critical contributor to AD progression. Additionally, mitochondrial biogenesis and proteostasis are critical for maintaining mitochondrial function and integrity. Impairments in these processes have been implicated in the progression of AD, further highlighting the multifaceted role of mitochondrial dysfunction in neurodegeneration. It further discusses the therapeutic potential of mitochondria-targeted strategies for AD drug development.
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spelling doaj-art-09d5885a705346cb91eebb8798dbad922025-08-20T03:16:52ZengBMCEuropean Journal of Medical Research2047-783X2025-05-0130112410.1186/s40001-025-02699-wThe role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapyXin Li0Ziyang Wu1Xiaying Si2Jing Li3Guode Wu4Manxia Wang5Department of Neurology, The Second Hospital & Clinical Medical School, Lanzhou UniversityThe Second Hospital & Clinical Medical School, Lanzhou UniversityDepartment of Psychiatry, The Second Hospital & Clinical Medical School, Lanzhou UniversityDepartment of Neurology, The Second Hospital & Clinical Medical School, Lanzhou UniversityDepartment of Neurology, The Second Hospital & Clinical Medical School, Lanzhou UniversityDepartment of Neurology, The Second Hospital & Clinical Medical School, Lanzhou UniversityAbstract Alzheimer's disease (AD) is a neurodegenerative disorder characterized by progressive cognitive decline, behavioral impairments, and psychiatric comorbidities. The pathogenesis of AD remains incompletely elucidated, despite advances in dominant hypotheses such as the β-amyloid (Aβ) cascade, tauopathy, cholinergic deficiency, and neuroinflammation mechanisms. However, these hypotheses inadequately explain the multifactorial nature of AD, which exposes limitations in our understanding of its mechanisms. Mitochondrial dysfunction is known to play a pivotal role in AD, and since patients exhibit intracellular mitochondrial dysfunction and structural changes in the brain at an early stage, correcting the imbalance of mitochondrial homeostasis and the cytopathological changes caused by it may be a potential target for early treatment of AD. Mitochondrial structural abnormalities accelerate AD pathogenesis. For instance, structural and functional alterations in the mitochondria-associated endoplasmic reticulum membrane (MAM) can disrupt intracellular Ca2⁺ homeostasis and cholesterol metabolism, consequently promoting Aβ accumulation. In addition, the overaccumulation of Aβ and hyperphosphorylated tau proteins can further damage neurons by disrupting mitochondrial integrity and mitophagy, thereby amplifying pathological aggregation and exacerbating neurodegeneration in AD. Furthermore, Aβ deposition and abnormal tau proteins can disrupt mitochondrial dynamics through dysregulation of fission/fusion proteins, leading to excessive mitochondrial fragmentation and subsequent dysfunction. Additionally, hyperphosphorylated tau proteins can impair mitochondrial transport, resulting in axonal dysfunction in AD. This article reviews the biological significance of mitochondrial structural morphology, dynamics, and mitochondrial DNA (mtDNA) instability in AD pathology, emphasizing mitophagy abnormalities as a critical contributor to AD progression. Additionally, mitochondrial biogenesis and proteostasis are critical for maintaining mitochondrial function and integrity. Impairments in these processes have been implicated in the progression of AD, further highlighting the multifaceted role of mitochondrial dysfunction in neurodegeneration. It further discusses the therapeutic potential of mitochondria-targeted strategies for AD drug development.https://doi.org/10.1186/s40001-025-02699-wAlzheimer’s diseaseMitophagyMitochondrial quality controlMitochondrial dynamicsTherapeutic targets
spellingShingle Xin Li
Ziyang Wu
Xiaying Si
Jing Li
Guode Wu
Manxia Wang
The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy
European Journal of Medical Research
Alzheimer’s disease
Mitophagy
Mitochondrial quality control
Mitochondrial dynamics
Therapeutic targets
title The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy
title_full The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy
title_fullStr The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy
title_full_unstemmed The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy
title_short The role of mitochondrial dysfunction in the pathogenesis of Alzheimer’s disease and future strategies for targeted therapy
title_sort role of mitochondrial dysfunction in the pathogenesis of alzheimer s disease and future strategies for targeted therapy
topic Alzheimer’s disease
Mitophagy
Mitochondrial quality control
Mitochondrial dynamics
Therapeutic targets
url https://doi.org/10.1186/s40001-025-02699-w
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