{"title":"Rewiring the OCD brain: Insights beyond cortico-striatal networks","authors":"Feyba Jijimon , Sonam Fathima Mehak , Zameel Mohammed , Samir Kumar Praharaj , Gireesh Gangadharan","doi":"10.1016/j.nbd.2025.107216","DOIUrl":"10.1016/j.nbd.2025.107216","url":null,"abstract":"<div><div>Obsessive-compulsive disorder (OCD) is a debilitating neuropsychiatric condition marked by recurrent obsessions and compulsions. Over the years, research has primarily focused on the cortico-striato-thalamo-cortical (CSTC) circuit and its neurochemical disruptions as an ideal circuit system for the development of treatment strategies. However, <!--> <!-->recent findings from neuroimaging studies and rodent research have highlighted the involvement of additional brain regions and network-level dysfunctions that are functionally linked to the CSTC loop. These findings suggest that the sole emphasis on the CSTC circuit is insufficient to address the clinical heterogeneity of the disorder. A broader perspective would therefore better capture the disorder's clinical heterogeneity and diverse cognitive and emotional deficits. Here, we review growing evidence for the role of extra-CSTC brain regions and associated neurotransmitter interactions in OCD, outlining potential pathways for novel therapeutic strategies.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107216"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145683466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Optic nerve glymphatic system: Physiological characterization, ischemia-induced changes, and neuroprotection in ischemic optic neuropathy","authors":"Yuning Zhang , Zidong Chen , Yufan Jiang, Qiaona Ye, Jicheng Lin, Xinyi Zhang, Deling Li, Qinyuan Hu, Yixiao Fu, Liling Liu, Yangfan Yang, Minbin Yu","doi":"10.1016/j.nbd.2025.107214","DOIUrl":"10.1016/j.nbd.2025.107214","url":null,"abstract":"<div><div>The glymphatic system facilitates cerebrospinal fluid (CSF) circulation and metabolic waste clearance in the central nervous system. Its alterations and neuroprotective potentials in cerebral disorders also have been confirmed. However, its role in the optic nerve and involvement in optic neuropathy remain unclear. Here, we utilized the methodology combining fluorescent tracer injection with tissue clearing to visualize and quantify bidirectional glymphatic flow in the optic nerve in mice. Under physiological conditions, low-molecular-weight CSF tracers entered the optic nerve, while intraocular tracers were efficiently cleared via glymphatic pathway—both processes could be temporarily suppressed by using TGN-020. Acute ischemia induced by unilateral common carotid artery occlusion (UCCAO) enhanced CSF accumulation in the optic nerve and impaired intraocular solute clearance. Administration of TGN-020 30 min before ischemia induction reduced CSF accumulation during the ischemic phase and preserved retinal and optic nerve structure and function after ischemia. These findings further reveal a widespread, highly active glymphatic system in the optic nerve. Ischemia-induced dynamic changes of glymphatic function may contribute to optic nerve injury, while usage of TGN-020 during acute ischemia holds certain therapeutic potential in ischemic optic neuropathy.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107214"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145687618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-22DOI: 10.1016/j.nbd.2025.107232
Xiao-Yue Xia , Zi-Qiang Huang , Hao-Hang Lin , Zhen-Yi Liu , Lin Zhang , Meng-Cheng Li , Yu-Qin Tu , Na-Ping Chen , Jun Ni , Qun-Lin Chen , Jian-Ping Hu , Shi-Rui Gan , Xin-Yuan Chen
{"title":"Diffusion along perivascular spaces as a marker for Glymphatic system impairment in spinocerebellar Ataxia type 3","authors":"Xiao-Yue Xia , Zi-Qiang Huang , Hao-Hang Lin , Zhen-Yi Liu , Lin Zhang , Meng-Cheng Li , Yu-Qin Tu , Na-Ping Chen , Jun Ni , Qun-Lin Chen , Jian-Ping Hu , Shi-Rui Gan , Xin-Yuan Chen","doi":"10.1016/j.nbd.2025.107232","DOIUrl":"10.1016/j.nbd.2025.107232","url":null,"abstract":"<div><div>Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disorder characterized by the accumulation of polyglutamylated ATXN3 protein within neurons, which can potentially compromise the integrity of the brain's glymphatic system. Our objective is to investigate whether glymphatic function is impaired in patients with SCA3 and its clinical relevance. This study recruited 129 SCA3 subjects, including 98 symptomatic (ataxic SCA3) and 31 presymptomatic (preataxic SCA3) individuals, along with 67 healthy controls (HCs). We calculated the index for diffusion tensor image analysis along the perivascular space (DTI-ALPS) across groups and examined its correlation with SCA3 clinical features. Except for the left cerebral hemisphere DTI-ALPS index showing no statistically significant difference between HC and preataxic SCA3, statistically significant differences in ALPS index were observed among the remaining three groups. The DTI-ALPS index decreased in the order HC group > preataxic SCA3 group > ataxic SCA3 group. The Ataxic SCA3 group exhibited a significantly lower DTI-ALPS index than the HC group. The mean DTI-ALPS index showed negative correlations with the Scale for the Assessment and Rating of Ataxia (SARA) scores and International Cooperative Ataxia Rating Scale (ICARS) scores. In this study, we demonstrate that glymphatic waste clearance is impaired in SCA3 and that the magnitude of ALPS-detected dysfunction parallels clinical burden. DTI-ALPS may serve as a potential indicator for evaluating glymphatic system alterations and disease.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107232"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828046","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-22DOI: 10.1016/j.nbd.2025.107239
Huaguang Yang , Yunfei Zha , Weiyin Vivian Liu , Zhi Wen , Gonghao Ling , Di Zhang , Wenbing Yang
{"title":"Linking the cerebellum posterior lobules atrophy to multiple system atrophy cognitive impairment","authors":"Huaguang Yang , Yunfei Zha , Weiyin Vivian Liu , Zhi Wen , Gonghao Ling , Di Zhang , Wenbing Yang","doi":"10.1016/j.nbd.2025.107239","DOIUrl":"10.1016/j.nbd.2025.107239","url":null,"abstract":"<div><h3>Background</h3><div>Cognitive impairments in multiple system atrophy (MSA) are linked to cerebellar dysfunction, but the role of specific posterior cerebellar lobules in domain-specific deficits remains unclear.</div></div><div><h3>Methods</h3><div>Structural MRI and cognitive assessments were conducted in 108 MSA patients (61 MSA-C, 47 MSA-P) and 53 healthy controls. Cerebellar gray matter (GM) volumes were segmented using the Spatially Unbiased Infratentorial Toolbox (SUIT). Multivariate regression models tested associations between posterior lobule volumes and cognitive domain scores.</div></div><div><h3>Results</h3><div>MSA patients showed significant GM atrophy in cerebellar subregions VI-VIII compared to controls, with greater reductions in MSA-C than MSA-P. Cognitive deficits were observed in visuospatial function, executive abilities, attention, and working memory across the cohort. Regression analyses revealed distinct patterns: In the overall MSA group, Crus II atrophy predicted visuospatial impairment, while Lobule VIIIa atrophy explained executive dysfunction. In MSA-C, Crus II atrophy remained a significant predictor of visuospatial deficits, but no cerebellar-predictive relationships emerged in MSA-P.</div></div><div><h3>Conclusions</h3><div>Distinct posterior cerebellar subregions contribute differentially to cognitive profiles in MSA. These findings highlight the role of cerebellar dysfunction in the pathophysiology of cognitive impairment across MSA subtypes.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107239"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145827982","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-18DOI: 10.1016/j.nbd.2025.107228
Manxi Xu , Yuanhao Li , Jixin Luan , Aocai Yang , Li Zhu , Ni Shu , Gaoxiang Ouyang , Yunjing Xue , Linwei Zhang , Guolin Ma
{"title":"Transcriptomic and neurotransmitter correlates of structure and function spatial variations patterns in spinocerebellar Ataxia","authors":"Manxi Xu , Yuanhao Li , Jixin Luan , Aocai Yang , Li Zhu , Ni Shu , Gaoxiang Ouyang , Yunjing Xue , Linwei Zhang , Guolin Ma","doi":"10.1016/j.nbd.2025.107228","DOIUrl":"10.1016/j.nbd.2025.107228","url":null,"abstract":"<div><h3>Background</h3><div>Spinocerebellar ataxia (SCA) is characterized by cortical structural and functional impairments, but the underlying biological mechanisms remain poorly understood.</div></div><div><h3>Methods</h3><div>We applied T1-weighted magnetic resonance imaging (MRI) and resting-state functional MRI (fMRI) to investigate cortical alterations in 49 SCA patients and 48 matched controls. Cortical thickness and amplitude of low-frequency fluctuations (ALFF) were quantified and compared with spatial maps of 19 neurotransmitter systems. Gene expression patterns were examined using partial least squares regression, followed by gene ontology analysis to identify associated biological processes.</div></div><div><h3>Results</h3><div>Both cortical thickness and ALFF emerged as sensitive biomarkers of cortical impairment in SCA. Thickness alterations were strongly associated with disruptions in cholinergic, serotonergic, and glutamatergic systems. ALFF alterations were linked to toxin-response and detoxification processes, with relevant gene expression enriched in astrocytes.</div></div><div><h3>Conclusion</h3><div>Our convergent imaging–transcriptomic analysis integrates direct molecular vulnerability and CTC-based transsynaptic degeneration, yielding a unified systems-level view of SCA. These findings go beyond correlation by prioritizing molecular pathways and non-neuronal cellular targets and enabling network-informed prediction of additional vulnerable regions.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107228"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145800560","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-17DOI: 10.1016/j.nbd.2025.107229
Kexin Duan , Caiyun Yang , Da Gao , Mengzhu Li , Mengxuan Chen , Haiyan Li , Weicong Zeng , Lijun Zhao , Meiling Zhu
{"title":"The multiple mechanisms of NR4A2 in neurological disorders and advances in targeted therapy research","authors":"Kexin Duan , Caiyun Yang , Da Gao , Mengzhu Li , Mengxuan Chen , Haiyan Li , Weicong Zeng , Lijun Zhao , Meiling Zhu","doi":"10.1016/j.nbd.2025.107229","DOIUrl":"10.1016/j.nbd.2025.107229","url":null,"abstract":"<div><div>Nuclear Receptor Subfamily 4 Group A Member 2 (NR4A2) is a transcription factor widely distributed in the central nervous system, playing a crucial regulatory role in various physiological and pathological processes, including dopaminergic neuronal differentiation, synaptic plasticity, neuroinflammation, immune responses, and addictive behaviors. Previous studies have primarily focused on exploring the relationship between NR4A2 and Parkinson's disease, with less attention given to the potential association between NR4A2 and other neurological disorders. In this context, this paper systematically reviews the current research on NR4A2 in Parkinson's disease, Alzheimer's disease, multiple sclerosis, psychiatric disorders, neurodevelopmental disorders (NDD), addictive behaviors, and stroke, revealing its multifaceted mechanisms in neurological disorders and elucidate its common and specific roles across different conditions. Furthermore, it also highlights cutting-edge strategies such as NR4A2 agonists, indirect NR4A2 regulators, and NR4A2-based cell replacement therapy or gene therapy, providing a comprehensive discussion of the progress and breakthroughs in NR4A2-targeted therapeutic approaches.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107229"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145794204","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-06DOI: 10.1016/j.nbd.2025.107219
Tae-Yong Choi , Aileen Gunawan , DaYeong Seo , Jinkyu Park , Eun Hee Ahn , Sang Won Suh , Marc V. Fuccillo , Kyuhyun Choi
{"title":"Applying biologically anchored subtypes to advance precision medicine in autism spectrum disorder","authors":"Tae-Yong Choi , Aileen Gunawan , DaYeong Seo , Jinkyu Park , Eun Hee Ahn , Sang Won Suh , Marc V. Fuccillo , Kyuhyun Choi","doi":"10.1016/j.nbd.2025.107219","DOIUrl":"10.1016/j.nbd.2025.107219","url":null,"abstract":"<div><div>Autism spectrum disorder (ASD) is heterogeneous at every level, from behavior to molecular pathways, limiting the value of subgrouping schemes built on surface phenotypes alone. We synthesize evidence that biologically anchored subtypes, defined by convergent genetics, developmental timing, and brain–body crosstalk, offer a tractable path to precision medicine. Leveraging advances in large-scale genomic resources and computational analytics, we propose a multi-axis framework: (i) genetic architecture spanning rare variants and polygenic load, (ii) developmental windows from mid-gestation to infancy divergence and regression, and (iii) brain–body interactions shaping plasticity and symptom expression. This framework enables mechanism-guided therapeutic strategies through biomarker-stratified enrollment, target-engagement readouts, and circuit-anchored outcomes. Preclinical platforms, genetically engineered mice and patient-derived induced pluripotent stem cells (iPSCs), demonstrate convergence onto limited synaptic and connectivity “neurotypes,” enabling causal links from gene to circuit to behavior and proof-of-concept rescue. We close with priorities: standardized multi-platform characterization, decision tools linking subtype labels to interventions, and stratified trials that co-report clinical and biological endpoints, with ethical guardrails to ensure early stratification expands opportunity while advancing individualized care.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107219"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145708625","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-13DOI: 10.1016/j.nbd.2025.107226
Brynna T. Webb , Hieu Trinh , Emily A. Breach , Kendall M. Foote , Erica Binelli , Geoffrey T. Swanson
{"title":"Pathological gain-of-function human variants in the GRIK2 kainate receptor gene cause wide-ranging behavioral dysfunction and seizures in mouse models","authors":"Brynna T. Webb , Hieu Trinh , Emily A. Breach , Kendall M. Foote , Erica Binelli , Geoffrey T. Swanson","doi":"10.1016/j.nbd.2025.107226","DOIUrl":"10.1016/j.nbd.2025.107226","url":null,"abstract":"<div><div><em>De novo</em> variants in a subset of ionotropic glutamate receptor (iGluR) genes cause nonsyndromic neurodevelopmental disorders (NDDs). Two recurrent variants in the kainate receptor (KAR) gene <em>GRIK2</em> result in the gain-of-function (GoF) substitutions p.Ala657Thr and p.Thr660Lys in a critical pore-forming domain of the GluK2 subunit. Disorders in individuals with these variants manifest as intellectual disability, developmental delay, motor impairments, and, in the case of p.Thr660Lys, epilepsy. To explore their pathogenicity and phenotypic consequences <em>in vivo</em>, we generated knock-in mouse models harboring orthologous <em>Grik2</em> mutations. Behavioral analyses revealed a range of developmental, motor, cognitive, and naturalistic behavior impairments in both lines, with the mouse model of the variant p.Thr660Lys, GluK2(T660K), exhibiting more severe phenotypes, consistent with clinical observations in humans. GluK2(T660K) mice also display interictal EEG abnormalities and handling-induced seizures. These models establish the first <em>in vivo</em> platforms for dissecting the underlying mechanisms of NDDs caused by GoF mutations in the GluK2 KAR subunit and represent crucial tools for therapeutic development.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107226"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757153","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cerebrospinal fluid α-synuclein seed amplification assay positivity is associated with increased plasma neurofilament light chain in Alzheimer's disease","authors":"Giovanni Bellomo PhD , Andrea Toja MD , Lorenzo Gaetani MD, PhD , Giovanna Nardi MD , Federico Paolini Paoletti MD, PhD , Davide Chiasserini PhD , Lucilla Parnetti MD, PhD","doi":"10.1016/j.nbd.2025.107234","DOIUrl":"10.1016/j.nbd.2025.107234","url":null,"abstract":"<div><h3>Background</h3><div>Synucleinopathy is the most common co-pathology in Alzheimer's disease (AD) and can be detected in vivo using cerebrospinal fluid (CSF) alpha-synuclein seed amplification assays (synSAA). CSF synSAA positivity has been linked to worse clinical outcome. This study investigated whether synSAA positivity is also associated with levels of plasma neurofilament light chain (NfL), a sensitive, unspecific marker of neurodegeneration.</div></div><div><h3>Methods</h3><div>We retrospectively analyzed a cohort of 240 individuals across the AD clinical continuum who had undergone clinical, neuropsychological, and biomarker assessments including CSF synSAA. Analyses involving plasma NfL were conducted in the subgroup with available plasma samples collected at the time of lumbar puncture (<em>n</em> = 166). One-way and two-way ANOVA were used to compare log-transformed NfL levels among AD stages, according to synSAA status. Logistic regression models examined associations between log-transformed NfL and synSAA status, adjusting for age and AD stage.</div></div><div><h3>Findings</h3><div>No significant age difference was found between synSAA-positive and negative groups. Plasma NfL levels were significantly higher in synSAA-positive individuals. This association remained significant after adjusting for age and AD clinical stage.</div></div><div><h3>Interpretation</h3><div>In AD patients, CSF synSAA positivity is associated with increase of plasma NfL levels along the AD clinical continuum. This finding supports the knowledge that synuclein co-pathology represents a contributive factor of neurodegeneration in AD patients.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107234"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Neurobiology of DiseasePub Date : 2026-01-01Epub Date: 2025-12-13DOI: 10.1016/j.nbd.2025.107224
Haoran Wang , Yuanzheng Qiao , Haimin Lu , Xitong Bo , Fuxiang Chen , Niu Pu , Yilong Zhou , Qiong Cheng
{"title":"Central integration mechanisms of neurovascular unit dysfunction and novel synergistic therapeutic strategies","authors":"Haoran Wang , Yuanzheng Qiao , Haimin Lu , Xitong Bo , Fuxiang Chen , Niu Pu , Yilong Zhou , Qiong Cheng","doi":"10.1016/j.nbd.2025.107224","DOIUrl":"10.1016/j.nbd.2025.107224","url":null,"abstract":"<div><div>The neurovascular unit (NVU) is a highly integrated multicellular complex composed of neurons, astrocytes, microglia, brain microvascular endothelial cells (BMECs), pericytes, and the extracellular matrix (ECM). It forms the structural and functional basis of the blood-brain barrier (BBB) and is pivotal for maintaining the homeostasis of the brain. Traditional neuroprotective strategies targeting individual cell types have shown limited efficacy in central nervous system (CNS) diseases, mainly due to the neglect of intricate intercellular crosstalk within the NVU. In this review, we first systematically summarize the core mechanisms by which the NVU functional unit causes NVU dysfunction in representative acute CNS injuries (ischemic/hemorrhagic stroke, traumatic brain injury), neurodegenerative diseases (Alzheimer's disease, Parkinson's disease, multiple sclerosis), and systemic diseases (diabetic encephalopathy, depression). Based on this, we innovatively summarize and clarify six major cross-disease pathological mechanisms of NVU dysfunction, including intercellular communication disorders, abnormal epigenetic modifications, microbiome-NVU interaction dysregulation, metabolic reprogramming dysfunction, neuroimmune-vascular coupling imbalance, and mechanical microenvironment imbalance. Additionally, we integrate emerging NVU models (co-culture systems, organoids, microfluidic chips, 3D bioprinting) with multi-omics technologies to establish a cross-scale dynamic research paradigm, and propose multicomponent coordinated regulatory strategies for NVU-targeted therapies. This framework aims to expand the understanding of NVU-centered pathological processes across diverse CNS diseases and provides a novel theoretical basis for precise therapeutic interventions, thereby bridging the gap between basic research and clinical translation.</div></div>","PeriodicalId":19097,"journal":{"name":"Neurobiology of Disease","volume":"218 ","pages":"Article 107224"},"PeriodicalIF":5.6,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}