entity

ASO

Entity Detail — Knowledge Graph Node

Understanding Entity Pages

This page aggregates everything SciDEX knows about ASO: 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.

5Connections
20Hypotheses
3Analyses
4Outgoing
1Incoming

Wiki Pages (21)

Knowledge base pages for this entity

Canonical Page

GTX-102 (GeneTx/Ultragenyx) — Angelman Syndrome Phase 1/2 Trial

clinical · 1741 words

Personalized Treatment Plan — Atypical Parkinsonism (CBS/PSP)

therapeutic · 15794 words

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

therapeutic · 15567 words

Progressive Supranuclear Palsy (PSP)

disease · 12907 words

Corticobasal Syndrome (CBS)

disease · 11374 words

Novel Therapy Index

idea · 10647 words

Outgoing (4)

TargetRelationTypeStr
SNCAinhibitsgene0.87
ALPHA_SYNUCLEINinhibitsprotein0.87
TDP-43targetsprotein0.75
PARKINSONS_DISEASEtreatsdisease0.64

Incoming (1)

SourceRelationTypeStr
TfR1bindsreceptor0.90

Targeting Hypotheses (20)

Hypotheses where this entity is a therapeutic target

HypothesisScoreDiseaseAnalysis
Closed-loop transcranial focused ultrasound to restore hippo 0.709 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop focused ultrasound targeting EC-II SST interneur 0.697 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop transcranial focused ultrasound with 40Hz gamma 0.689 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop transcranial focused ultrasound to restore hippo 0.633 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop transcranial focused ultrasound targeting EC-II 0.633 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop focused ultrasound targeting CA1 PV interneurons 0.614 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop transcranial focused ultrasound to restore hippo 0.614 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop transcranial focused ultrasound targeting EC-II 0.612 Alzheimer's disease Circuit-level neural dynamics in neurode
ASO-Mediated Exon Skipping to Restore FUS-TAZ Chaperone Axis 0.606 neurodegeneration How does FUS loss-of-function in TAZ reg
Microglial AIM2 Inflammasome as the Primary Driver of TDP-43 0.601 neurodegeneration What are the mechanisms by which gut mic
Astrocyte-Intrinsic NLRP3 Inflammasome Activation by Alpha-S 0.599 neurodegeneration What are the mechanisms by which gut mic
Microbial Inflammasome Priming Prevention 0.584 neurodegeneration What are the mechanisms by which gut mic
Mitochondrial DAMPs-Driven AIM2 Inflammasome Activation in N 0.582 neurodegeneration What are the mechanisms by which gut mic
Calcium-Dysregulated mPTP Opening as an Alternative mtDNA Re 0.581 neurodegeneration What are the mechanisms by which gut mic
Mitochondrial DNA-Driven AIM2 Inflammasome Activation in Neu 0.580 neurodegeneration What are the mechanisms by which gut mic
TDP-43 Cryptic Exon–Targeted ASOs to Restore Hippocampal Gam 0.577 neurodegeneration RNA binding protein dysregulation across
Closed-loop focused ultrasound targeting EC-II PV interneuro 0.568 Alzheimer's disease Circuit-level neural dynamics in neurode
Real-time gamma-guided transcranial focused ultrasound targe 0.538 Alzheimer's disease Circuit-level neural dynamics in neurode
Closed-loop transcranial focused ultrasound targeting CA1 PV 0.508 Alzheimer's disease Circuit-level neural dynamics in neurode
Early Proteasome Restoration Therapy 0.496 neurodegeneration Gene expression changes in aging mouse b

Mentioning Analyses (3)

Scientific analyses that reference this entity

What is the optimal BBB opening duration and spatial precision to maximize thera

neuropharmacology | 2026-04-11 | 0 hypotheses

What are the conserved secondary structures in dilncRNAs that enable selective t

molecular biology | 2026-04-10 | 0 hypotheses

Lysosomal dysfunction and cathepsin leakage in Alzheimer disease progression

neuroscience | 2026-04-04 | 0 hypotheses

Related Papers (10)

Scientific publications cited in analyses involving this entity

Title & PMIDAuthorsJournalYearCitations
Gray matter structural and molecular signatures in Alzheimer's disease and late- [PMID:41419068] Yang S, Wang X, Yan Y, Zhang J, Wei Y et J Affect Disord 2026 0
Cytomegalic parvalbumin neurons in fetal cases of hemimegalencephaly. [PMID:39973610] Gelot AB, Draia-Nicolau TO, Mathieu R, S Epilepsia 2025 0
Brain connectivity and transcriptional changes induced by rTMS in first-episode [PMID:40274783] Guan M, Xie Y, Wang Z, Miao Y, Li X et a Transl Psychiatry 2025 0
Single-cell RNA sequencing of adult primate neocortex reveals the regulatory dyn [PMID:40385068] Zhang C, Xie Z, Wang N Am J Transl Res 2025 0
Human stem cell-derived GABAergic interneuron development reveals early emergenc [PMID:40651475] Bershteyn M, Zhou H, Fuentealba L, Chen Neuron 2025 0
Targeting SARM1 improves autophagic stress-induced axonal neuropathy. [PMID:37561040] Kim HR, Lee HJ, Jeon Y, Jang SY, Shin YK Autophagy 2024 0
Integrated multimodal cell atlas of Alzheimer's disease. [PMID:39402379] Gabitto MI, Travaglini KJ, Rachleff VM, Nat Neurosci 2024 0
Identification of early Alzheimer's disease subclass and signature genes based o [PMID:39650656] Wang W, Lu J, Pan N, Zhang H, Dai J et a Front Immunol 2024 0
Meta-proteomics for the discovery of protein biomarkers of beef tenderness: An o [PMID:31882086] Picard B, Gagaoua M Food Res Int 2020 0
Identification of epilepsy-associated neuronal subtypes and gene expression unde [PMID:33028830] Pfisterer U, Petukhov V, Demharter S, Me Nat Commun 2020 0