Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model

Abstract Blood-brain barrier (BBB) dysfunction is an early pathological hallmark of Alzheimer’s disease (AD), occurring prior to amyloid-β (Aβ) accumulation. A key factor contributing to BBB damage in AD is the loss of endothelial expression of low-density lipoprotein receptor-related protein 1 (LRP...

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Main Authors: Ting-Ting Pan, Yan-Yun Sun, Yi-Fan Shi, Mei Zhao, Naveed Ullah Khan, Hai-Yan Chen, Wen-Li Ji, Jie Li, Liang Han, Quan-Hong Ma
Format: Article
Language:English
Published: BMC 2025-08-01
Series:Alzheimer’s Research & Therapy
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Online Access:https://doi.org/10.1186/s13195-025-01840-5
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author Ting-Ting Pan
Yan-Yun Sun
Yi-Fan Shi
Mei Zhao
Naveed Ullah Khan
Hai-Yan Chen
Wen-Li Ji
Jie Li
Liang Han
Quan-Hong Ma
author_facet Ting-Ting Pan
Yan-Yun Sun
Yi-Fan Shi
Mei Zhao
Naveed Ullah Khan
Hai-Yan Chen
Wen-Li Ji
Jie Li
Liang Han
Quan-Hong Ma
author_sort Ting-Ting Pan
collection DOAJ
description Abstract Blood-brain barrier (BBB) dysfunction is an early pathological hallmark of Alzheimer’s disease (AD), occurring prior to amyloid-β (Aβ) accumulation. A key factor contributing to BBB damage in AD is the loss of endothelial expression of low-density lipoprotein receptor-related protein 1 (LRP1). Endothelial LRP1 is crucial for maintaining BBB integrity and facilitating the transcytosis of Aβ across the BBB for peripheral clearance. However, LRP1 is also expressed in other neural cell types, such as neurons, where it paradoxically promotes Aβ generation and tau propagation. These dual roles of LRP1 for different cell types present a challenge for developing effective AD therapy targeting LRP1. Simvastatin (SIM), an HMG-CoA reductase inhibitor, has been shown to induce compensatory upregulation of LRP1 expression. To harness this potential, we designed SIM-loaded Angiopep-2-anchored nanoparticles (S@A-NPs) that can be effectively internalized by endothelial cells. Our findings demonstrate that intravenous (IV) injection with S@A-NPs upregulates endothelial LRP1 expression level, repairs BBB damage, attenuates Aβ accumulation, mitigates neurodegeneration, and ultimately preserves cognitive function in APP/PS1 mice. These results highlight the potential of endothelial delivery of SIM via nanoparticles to attenuate AD pathogenesis. Our study proposes a novel therapeutic strategy for AD by leveraging nanoparticle-mediated drug delivery.
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institution Kabale University
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publishDate 2025-08-01
publisher BMC
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series Alzheimer’s Research & Therapy
spelling doaj-art-3b8d9ba4d19e4dce8c0228a8fbe2618d2025-08-24T11:12:01ZengBMCAlzheimer’s Research & Therapy1758-91932025-08-0117112410.1186/s13195-025-01840-5Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse modelTing-Ting Pan0Yan-Yun Sun1Yi-Fan Shi2Mei Zhao3Naveed Ullah Khan4Hai-Yan Chen5Wen-Li Ji6Jie Li7Liang Han8Quan-Hong Ma9Department of Neurology and Clinical Research Center of Neurological Disease, the Second Affiliated Hospital of Soochow UniversityDepartment of Neurology and Clinical Research Center of Neurological Disease, the Second Affiliated Hospital of Soochow UniversityDepartment of Neurology and Clinical Research Center of Neurological Disease, the Second Affiliated Hospital of Soochow UniversityJiangsu Key Laboratory of Translational Research and Therapy for Neuro-Psycho- Diseases and College of Pharmaceutical Sciences, Soochow UniversityJiangsu Key Laboratory of Translational Research and Therapy for Neuro-Psycho- Diseases and College of Pharmaceutical Sciences, Soochow UniversityJiangsu Key Laboratory of Translational Research and Therapy for Neuro-Psycho- Diseases and College of Pharmaceutical Sciences, Soochow UniversityDepartment of Neurology and Clinical Research Center of Neurological Disease, the Second Affiliated Hospital of Soochow UniversityDepartment of General Practice, The First Affiliated Hospital of Soochow University, Soochow UniversityJiangsu Key Laboratory of Translational Research and Therapy for Neuro-Psycho- Diseases and College of Pharmaceutical Sciences, Soochow UniversityDepartment of Neurology and Clinical Research Center of Neurological Disease, the Second Affiliated Hospital of Soochow UniversityAbstract Blood-brain barrier (BBB) dysfunction is an early pathological hallmark of Alzheimer’s disease (AD), occurring prior to amyloid-β (Aβ) accumulation. A key factor contributing to BBB damage in AD is the loss of endothelial expression of low-density lipoprotein receptor-related protein 1 (LRP1). Endothelial LRP1 is crucial for maintaining BBB integrity and facilitating the transcytosis of Aβ across the BBB for peripheral clearance. However, LRP1 is also expressed in other neural cell types, such as neurons, where it paradoxically promotes Aβ generation and tau propagation. These dual roles of LRP1 for different cell types present a challenge for developing effective AD therapy targeting LRP1. Simvastatin (SIM), an HMG-CoA reductase inhibitor, has been shown to induce compensatory upregulation of LRP1 expression. To harness this potential, we designed SIM-loaded Angiopep-2-anchored nanoparticles (S@A-NPs) that can be effectively internalized by endothelial cells. Our findings demonstrate that intravenous (IV) injection with S@A-NPs upregulates endothelial LRP1 expression level, repairs BBB damage, attenuates Aβ accumulation, mitigates neurodegeneration, and ultimately preserves cognitive function in APP/PS1 mice. These results highlight the potential of endothelial delivery of SIM via nanoparticles to attenuate AD pathogenesis. Our study proposes a novel therapeutic strategy for AD by leveraging nanoparticle-mediated drug delivery.https://doi.org/10.1186/s13195-025-01840-5SimvastatinNanoparticlesAlzheimer’s diseaseLRP1BBBAmyloid-β
spellingShingle Ting-Ting Pan
Yan-Yun Sun
Yi-Fan Shi
Mei Zhao
Naveed Ullah Khan
Hai-Yan Chen
Wen-Li Ji
Jie Li
Liang Han
Quan-Hong Ma
Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model
Alzheimer’s Research & Therapy
Simvastatin
Nanoparticles
Alzheimer’s disease
LRP1
BBB
Amyloid-β
title Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model
title_full Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model
title_fullStr Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model
title_full_unstemmed Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model
title_short Endothelial delivery of simvastatin by LRP1-targeted nanoparticles ameliorates pathogenesis of alzheimer’s disease in a mouse model
title_sort endothelial delivery of simvastatin by lrp1 targeted nanoparticles ameliorates pathogenesis of alzheimer s disease in a mouse model
topic Simvastatin
Nanoparticles
Alzheimer’s disease
LRP1
BBB
Amyloid-β
url https://doi.org/10.1186/s13195-025-01840-5
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