drug

AAV

Entity Detail — Knowledge Graph Node

Understanding Entity Pages

This page aggregates everything SciDEX knows about AAV: its mechanistic relationships (Knowledge Graph edges), hypotheses targeting it, analyses mentioning it, and supporting scientific papers. The interactive graph below shows its immediate neighbors. All content is AI-synthesized from peer-reviewed literature.

18Connections
10Hypotheses
3Analyses
18Outgoing
0Incoming
6Experiments
3Debates

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💊 Drug Info
NameAAV
Key Genes/ProteinsAPOE, BDNF, Cas9, GDNF, NDNF
Related DiseasesMacaque, Rodents
Related PathwaysDescending Motor Pathways
Linked Hypotheses10 hypotheses

Wiki Pages (17)

Knowledge base pages for this entity

Canonical Page

PBKR03

entity · 3476 words

AAV Gene Therapy for Neurodevelopmental Epilepsy — Competitive Landscape & Delivery Alternatives

therapeutic · 15567 words

AAV Gene Therapy Vectors for Neurodegeneration

mechanism · 2941 words

AAV-LRRK2 IND-Enabling Study Design

experiment · 1843 words

AAV-Delivered RNA Targeting Therapy for Neurodegeneration

idea · 1497 words

AAV Gene Therapy Vectors for Neurodegenerative Diseases

therapeutic · 1465 words

Pathway Diagram

graph TD
    AAV["AAV"] -->|"associated"| Rodents["Rodents"]
    style Rodents fill:#1a2a3a,stroke:#ffd54f,stroke-width:1px,color:#fff
    AAV["AAV"] -->|"associated"| Macaque["Macaque"]
    style Macaque fill:#1a2a3a,stroke:#ffd54f,stroke-width:1px,color:#fff
    AAV["AAV"] -->|"targets"| NDNF["NDNF"]
    style NDNF fill:#1a2a3a,stroke:#4fc3f7,stroke-width:1px,color:#fff
    AAV["AAV"] -->|"targets"| Brain["Brain"]
    style Brain fill:#1a2a3a,stroke:#4fc3f7,stroke-width:1px,color:#fff
    AAV["AAV"] -->|"transports"| BDNF["BDNF"]
    style BDNF fill:#1a2a3a,stroke:#4fc3f7,stroke-width:1px,color:#fff
    AAV["AAV"] -->|"transports"| APOE["APOE"]
    style APOE fill:#1a2a3a,stroke:#ce93d8,stroke-width:1px,color:#fff
    AAV["AAV"] -->|"transports"| GDNF["GDNF"]
    style GDNF fill:#1a2a3a,stroke:#4fc3f7,stroke-width:1px,color:#fff
    style AAV fill:#1b5e20,stroke:#4fc3f7,stroke-width:2px,color:#e0e0e0,font-weight:bold

Outgoing (18)

TargetRelationTypeStr
Rodentsassociated_withphenotype0.90
Macaqueassociated_withphenotype0.90
NDNFtargetsprotein0.90
Rodentsexpressed_inphenotype0.90
Macaqueexpressed_inphenotype0.90

Incoming (0)

SourceRelationTypeStr
No incoming edges

Targeting Hypotheses (10)

Hypotheses where this entity is a therapeutic target

HypothesisScoreDiseaseAnalysis
AAV-PHP.eB-Mediated Microglial IGFBPL1 Expression 0.768 drug-delivery Can IGFBPL1 therapeutics effectively cro
AAV-Mediated APOE2/APOE3 Gene Delivery to Convert APOE Genot 0.700 neurodegeneration APOE4 targeting in neurodegeneration
TREM2 R47H Variant Correction — AAV-Mediated Rescue of Commo 0.631 alzheimers TREM2 agonism vs antagonism in DAM micro
CX3CR1-Targeted HDAC3 Knockdown in Microglia via AAV9 Delive 0.620 pharmacology How can CNS-selective HDAC/DNMT inhibito
AAV-mediated RGS6 re-expression in SNpc after pathology onse 0.530 neurodegeneration Does RGS6 upregulation or D2 autorecepto
AAV-PHP.eB-Medium OSK Expression Reverses Cortical Neuronal 0.520 neurodegeneration epigenetic reprogramming aging neurons
AAV-Mediated RGS6 Overexpression in Substantia Nigra Parvoce 0.424 neurodegeneration Does RGS6 upregulation or D2 autorecepto
FUS-Enhanced AAV-PHP.eB Delivery for Microglial IGFBPL1 Expr 0.380 drug-delivery Can IGFBPL1 therapeutics effectively cro
AAV-PHP.eB-Mediated Astrocytic IGFBPL1 Expression 0.359 drug-delivery Can IGFBPL1 therapeutics effectively cro
FUS-Enhanced AAV-PHP.eB Delivery of IGFBPL1 to Microglia 0.338 drug-delivery Can IGFBPL1 therapeutics effectively cro

Mentioning Analyses (3)

Scientific analyses that reference this entity

What delivery mechanisms can achieve therapeutically relevant CRISPR concentrati

drug delivery | 2026-04-10 | 0 hypotheses

CRISPR-based therapeutic approaches for neurodegenerative diseases

neurodegeneration | 2026-04-03 | 14 hypotheses Top: 0.850

CRISPR-based therapeutic approaches for neurodegenerative diseases

neurodegeneration | 2026-04-02 | 0 hypotheses

Experiments (6)

Experimental studies targeting or related to this entity

ExperimentTypeDiseaseScoreFeasibilityModelStatusEst. Cost
CHI3L1 variant association with AD progression in human patients exploratory Alzheimer's disease 0.850 0.00 human patients proposed N/A
AAV-LRRK2 Gene Therapy IND-Enabling Study Design clinical Parkinson's Disease 0.400 0.50 human proposed $5,460,000
AAV-LRRK2 IND-Enabling Study Design clinical Parkinson's Disease 0.400 0.50 human proposed $7,500,000
AAV Serotype Comparison for LRRK2 Knockdown in PD Gene Therapy validation Parkinson's Disease 0.400 0.50 mouse proposed $280,000
AAV Serotype Comparison for LRRK2 Knockdown in PD validation Parkinson's Disease 0.400 0.50 mouse proposed $280,000
Gene Therapy: AAV Serotype Comparison for LRRK2 Knockdown validation Parkinson's Disease 0.400 0.50 mouse proposed $280,000

Related Papers (16)

Scientific publications cited in analyses involving this entity

Title & PMIDAuthorsJournalYearCitations
The gain-of-function TREM2-T96K mutation increases risk for Alzheimer's disease [PMID:41109213] Pilat DJ, Le H, Prokopenko D, Lin CJ, Ei Neuron 2026 0
Role of TREM2 in neuroinflammation. [PMID:41213496] Abdulkhaliq AA, Alasiri G, Almoghrabi Y, Exp Neurol 2026 0
Synergistic potential of TREM2 agonists and exercise training in Alzheimer's dis [PMID:41494649] Zhang J, St Pierre Schneider B, Muguerza Am J Physiol Endocrinol Metab 2026 0
Armored macrophage-targeted CAR-T cells reset and reprogram the tumor microenvir [PMID:41576929] Mateus-Tique J, Lakshmi A, Singh B, Iyer Cancer Cell 2026 0
Peripheral cancer attenuates amyloid pathology in Alzheimer's disease via cystat [PMID:41576952] Li X, Tang X, Zeng J, Duan L, Hou Z et a Cell 2026 0
TREM2 expression level is critical for microglial state, metabolic capacity and [PMID:41580393] Feiten AF, Dahm K, Schlepckow K, van Len Nat Commun 2026 0
Large-scale single-cell analysis and in silico perturbation reveal dynamic evolu [PMID:41617928] Xia P, Shuang S, Fu D, Liu L, Yang D et NPJ Precis Oncol 2026 0
Dissecting genetic and immune drivers of heterogeneous responses to neoadjuvant [PMID:41720086] Zhao Q, Huang R, Wang C, Hu J, Nie R et Cancer Cell 2026 0
TREM2 in neurodegeneration and diseases. [PMID:41792456] ["Abdulkhaliq A", "Alasiri G", "Kim B", Molecular psychiatry 2026 0
Alzheimer's Disease as a Disorder of Neuroimmune Dysregulation. [PMID:41745721] Aranda-Abreu GE, Rojas-Durán F, Hernánde Neurology international 2026 0
Molecular mechanism of Alzheimer's disease using integrated multi-omics. [PMID:41907842] Alhusaini M, Mussa BM, Ilce BY, Alhaj H, Frontiers in aging neuroscienc 2026 0
Viral and non-viral cellular therapies for neurodegeneration. [PMID:41585268] ["Srivastav Jyotsna", "Sharma Sachin"] Frontiers in medicine 2025 0
Focused ultrasound-mediated APOE4 knockdown in mouse brain. [PMID:40665471] Zheng K, Tsitsos FN, Batts AJ, Ji R, Nur Alzheimer's & dementia : the j 2025 0
Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity. [PMID:39893321] Gumusgoz E, Kasiri S, Youssef I, Verma M Gene therapy 2025 0
IGFBPL1 is a master driver of microglia homeostasis and resolution of neuroinfla [PMID:37527036] Pan L, Cho KS, Wei X, Xu F, Lennikov A, Cell reports 2023 0
Incisionless targeted adeno-associated viral vector delivery to the brain by foc [PMID:36152518] Unknown EBioMedicine 2022 0

Debates (3)

Multi-agent debates referencing this entity

All participants identified BBB penetration as a critical bottleneck, but no exi

closed · Rounds: 4 · Score: 0.50 · 2026-04-20

Evaluate the potential of CRISPR/Cas9 and related gene editing technologies for

closed · Rounds: 4 · Score: 0.95 · 2026-04-03

Evaluate the potential of CRISPR/Cas9 and related gene editing technologies for

closed · Rounds: 4 · Score: 0.90 · 2026-04-02

Related Research

Hypotheses and analyses mentioning AAV in their description or question text

AAV-PHP.eB-Medium OSK Expression Reverses Cortical Neuronal Epigenetic Age Witho

Score: 0.520 · neurodegeneration · 2026-04-22

## Mechanistic Overview AAV-PHP.eB-Medium OSK Expression Reverses Cortical Neuronal Epigenetic Age Without Altering Glia

AAV-Mediated RGS6 Overexpression in Substantia Nigra Parvocellular Neurons

Score: 0.424 · neurodegeneration · 2026-04-18

## Mechanistic Overview AAV-Mediated RGS6 Overexpression in Substantia Nigra Parvocellular Neurons starts from the claim

FUS-Enhanced AAV-PHP.eB Delivery for Microglial IGFBPL1 Expression

Score: 0.380 · drug-delivery · 2026-04-27

This hybrid approach combines focused ultrasound (FUS) microbubble-mediated blood-brain barrier (BBB) disruption with AA

AAV-PHP.eB-Mediated Astrocytic IGFBPL1 Expression

Score: 0.359 · drug-delivery · 2026-04-26

**Molecular Mechanism and Rationale** The AAV-PHP.eB-mediated delivery of IGFBPL1 to astrocytes leverages the neurotrop

FUS-Enhanced AAV-PHP.eB Delivery of IGFBPL1 to Microglia

Score: 0.338 · drug-delivery · 2026-04-26

This hypothesis combines focused ultrasound (FUS) microbubble-mediated blood-brain barrier disruption with AAV-PHP.eB vi