Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model

Abstract Characteristic cerebral pathological changes of Alzheimer’s disease (AD) such as glucose hypometabolism or the accumulation of cleavage products of the amyloid precursor protein (APP), known as Aβ peptides, lead to sustained endoplasmic reticulum (ER) stress and neurodegeneration. To preser...

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Main Authors: Ylauna Christine Mégane Penalva, Sandra Paschkowsky, Jingyun Yang, Sherilyn Junelle Recinto, Jessica K. Cinkornpumin, Marina Ruelas, Bin Xiao, Albert Nitu, Sin Young Kwon, Helen Yee-Li Wu, Hans Markus Munter, Bernadeta Michalski, Margaret Fahnestock, William A. Pastor, David A. Bennett, Lisa Marie Munter
Format: Article
Language:English
Published: Nature Publishing Group 2025-04-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-025-07579-z
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author Ylauna Christine Mégane Penalva
Sandra Paschkowsky
Jingyun Yang
Sherilyn Junelle Recinto
Jessica K. Cinkornpumin
Marina Ruelas
Bin Xiao
Albert Nitu
Sin Young Kwon
Helen Yee-Li Wu
Hans Markus Munter
Bernadeta Michalski
Margaret Fahnestock
William A. Pastor
David A. Bennett
Lisa Marie Munter
author_facet Ylauna Christine Mégane Penalva
Sandra Paschkowsky
Jingyun Yang
Sherilyn Junelle Recinto
Jessica K. Cinkornpumin
Marina Ruelas
Bin Xiao
Albert Nitu
Sin Young Kwon
Helen Yee-Li Wu
Hans Markus Munter
Bernadeta Michalski
Margaret Fahnestock
William A. Pastor
David A. Bennett
Lisa Marie Munter
author_sort Ylauna Christine Mégane Penalva
collection DOAJ
description Abstract Characteristic cerebral pathological changes of Alzheimer’s disease (AD) such as glucose hypometabolism or the accumulation of cleavage products of the amyloid precursor protein (APP), known as Aβ peptides, lead to sustained endoplasmic reticulum (ER) stress and neurodegeneration. To preserve ER homeostasis, cells activate their unfolded protein response (UPR). The rhomboid-like-protease 4 (RHBDL4) is an enzyme that participates in the UPR by targeting proteins for proteasomal degradation. We demonstrated previously that RHBDL4 cleaves APP in HEK293T cells, leading to decreased total APP and Aβ. More recently, we showed that RHBDL4 processes APP in mouse primary mixed cortical cultures as well. Here, we aim to examine the physiological relevance of RHBDL4 in the brain. We first found that brain samples from AD patients and an AD mouse model (APPtg) showed increased RHBDL4 mRNA and protein expression. To determine the effects of RHBDL4’s absence on APP physiology in vivo, we crossed APPtg mice to a RHBDL4 knockout (R4−/−) model. RHBDL4 deficiency in APPtg mice led to increased total cerebral APP and amyloidogenic processing when compared to APPtg controls. Contrary to expectations, as assessed by cognitive tests, RHBDL4 absence rescued cognition in 5-month-old female APPtg mice. Informed by unbiased RNA-seq data, we demonstrated in vitro and in vivo that RHBDL4 absence leads to greater levels of active β-catenin due to decreased proteasomal clearance. Decreased β-catenin activity is known to underlie cognitive defects in APPtg mice and AD. Our work suggests that RHBDL4’s increased expression in AD, in addition to regulating APP levels, leads to aberrant degradation of β-catenin, contributing to cognitive impairment.
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spelling doaj-art-a08bd5facd4b49f7bdca2696bb5feb6e2025-08-20T02:11:47ZengNature Publishing GroupCell Death and Disease2041-48892025-04-0116111510.1038/s41419-025-07579-zLoss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse modelYlauna Christine Mégane Penalva0Sandra Paschkowsky1Jingyun Yang2Sherilyn Junelle Recinto3Jessica K. Cinkornpumin4Marina Ruelas5Bin Xiao6Albert Nitu7Sin Young Kwon8Helen Yee-Li Wu9Hans Markus Munter10Bernadeta Michalski11Margaret Fahnestock12William A. Pastor13David A. Bennett14Lisa Marie Munter15Department of Pharmacology & Therapeutics, McGill UniversityDepartment of Pharmacology & Therapeutics, McGill UniversityRush Alzheimer’s Disease Center, Rush University Medical CenterDepartment of Pharmacology & Therapeutics, McGill UniversityDepartment of Biochemistry, McGill UniversityDepartment of Pharmacology & Therapeutics, McGill UniversityIntegrated Program in Neuroscience, McGill UniversityIntegrated Program in Neuroscience, McGill UniversityDepartment of Biochemistry, McGill UniversityDepartment of Pharmacology & Therapeutics, McGill UniversityDepartment of Human Genetics, McGill UniversityDepartment of Psychiatry and Behavioural Neurosciences, McMaster UniversityDepartment of Psychiatry and Behavioural Neurosciences, McMaster UniversityDepartment of Biochemistry, McGill UniversityRush Alzheimer’s Disease Center, Rush University Medical CenterDepartment of Pharmacology & Therapeutics, McGill UniversityAbstract Characteristic cerebral pathological changes of Alzheimer’s disease (AD) such as glucose hypometabolism or the accumulation of cleavage products of the amyloid precursor protein (APP), known as Aβ peptides, lead to sustained endoplasmic reticulum (ER) stress and neurodegeneration. To preserve ER homeostasis, cells activate their unfolded protein response (UPR). The rhomboid-like-protease 4 (RHBDL4) is an enzyme that participates in the UPR by targeting proteins for proteasomal degradation. We demonstrated previously that RHBDL4 cleaves APP in HEK293T cells, leading to decreased total APP and Aβ. More recently, we showed that RHBDL4 processes APP in mouse primary mixed cortical cultures as well. Here, we aim to examine the physiological relevance of RHBDL4 in the brain. We first found that brain samples from AD patients and an AD mouse model (APPtg) showed increased RHBDL4 mRNA and protein expression. To determine the effects of RHBDL4’s absence on APP physiology in vivo, we crossed APPtg mice to a RHBDL4 knockout (R4−/−) model. RHBDL4 deficiency in APPtg mice led to increased total cerebral APP and amyloidogenic processing when compared to APPtg controls. Contrary to expectations, as assessed by cognitive tests, RHBDL4 absence rescued cognition in 5-month-old female APPtg mice. Informed by unbiased RNA-seq data, we demonstrated in vitro and in vivo that RHBDL4 absence leads to greater levels of active β-catenin due to decreased proteasomal clearance. Decreased β-catenin activity is known to underlie cognitive defects in APPtg mice and AD. Our work suggests that RHBDL4’s increased expression in AD, in addition to regulating APP levels, leads to aberrant degradation of β-catenin, contributing to cognitive impairment.https://doi.org/10.1038/s41419-025-07579-z
spellingShingle Ylauna Christine Mégane Penalva
Sandra Paschkowsky
Jingyun Yang
Sherilyn Junelle Recinto
Jessica K. Cinkornpumin
Marina Ruelas
Bin Xiao
Albert Nitu
Sin Young Kwon
Helen Yee-Li Wu
Hans Markus Munter
Bernadeta Michalski
Margaret Fahnestock
William A. Pastor
David A. Bennett
Lisa Marie Munter
Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model
Cell Death and Disease
title Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model
title_full Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model
title_fullStr Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model
title_full_unstemmed Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model
title_short Loss of the APP regulator RHBDL4 preserves memory in an Alzheimer’s disease mouse model
title_sort loss of the app regulator rhbdl4 preserves memory in an alzheimer s disease mouse model
url https://doi.org/10.1038/s41419-025-07579-z
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