gene

TARDBP

Entity Detail — Knowledge Graph Node

Understanding Entity Pages

This page aggregates everything SciDEX knows about TARDBP: 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.

1238Connections

4Hypotheses

4Analyses

50Outgoing

50Incoming

Summary

TARDBP is a gene implicated in neurodegeneration research. Key relationships include: associated with, activates, interacts with. Associated with AD, ALI, ALS. Connected to 534 entities in the SciDEX knowledge graph.

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🧬 Gene Info

Gene Symbol	TARDBP
Full Name	TAR DNA Binding Protein 43
Chromosome	1p36
Protein Type	Transcription Factor
Target Class	Transcription Factor
Function	RNA-binding and transcription regulation, challenging for small molecule targeting
Mechanism of Action	RNA-binding and transcription regulation, challenging for small molecule targeting
Druggability	Low (0.43)
Clinical Stage	Phase III
Molecular Weight	43 kDa
Amino Acids	4477 aa
Pathways	Apoptosis, Autophagy, Immune Response, Oxidative Stress, Tdp-43
UniProt ID	A0A087WYY0
GeneCards	TARDBP
Human Protein Atlas	TARDBP
Associated Diseases	AD, ALI, ALS, ALS-FTD, Alzheimer
Known Drugs/Compounds	Transcribed Rnas, RAPAMYCIN, rapamycin
Interactions	NTN1, SNRNP70, NTN3, ATG10, PINK1, LC3
SciDEX Target	View Target Profile (7 clinical trials)
SciDEX Hypotheses	Cryptic Exon Silencing Restoration Cross-Seeding Prevention Strategy Glycine-Rich Domain Competitive Inhibition (+1 more)
KG Connections	1201 knowledge graph edges
Databases	GeneCards NCBI Gene HPA STRING

🧬 3D Structure: TARDBP — PDB 4BS2 Click to expand

Experimental structure from RCSB PDB | Powered by Mol*

Wiki Pages (21)

Knowledge base pages for this entity

Canonical Page

Pathway Diagram

graph TD
    A["SRPK1"] -->|"phosphorylates"| B["TARDBP/TDP-43"]
    C["PRMT1"] -->|"methylates"| B
    B -->|"binds RNA"| D["RNA Processing"]
    B -->|"regulates"| E["HNRNPA2B1"]
    B -->|"interacts"| F["NPM1"]
    D -->|"impaired"| G["Stress Granules"]
    G -->|"contains"| H["G3BP1"]
    B -->|"aggregates"| I["TDP-43 Proteinopathy"]
    J["PARP1"] -->|"modulates"| B
    K["HSPA1A"] -->|"chaperone activity"| B
    I -->|"leads to"| L["ALS/FTD"]
    I -->|"causes"| M["Neurodegeneration"]
    G -->|"dysfunction"| M

Outgoing (729)

Target	Relation	Type	Str
TDP-43	associated_with	protein	1.00
TAU	associated_with	protein	1.00
FTD	associated_with	disease	1.00
DEMENTIA	associated_with	disease	1.00
FRONTOTEMPORAL DEMENTIA	associated_with	disease	1.00
NEURON	associated_with	cell_type	1.00
CORTEX	associated_with	brain_region	1.00
Tdp-43 Aggregation	involved_in	process	0.98
Loss Of Function	causes	phenotype	0.98
Frontotemporal Lobar Degeneration with TDP-43 Type C	implicated_in	disease	0.98
UNC13A	regulates	gene	0.95
Ftld-Tdp	component_of	disease	0.95
NEAT1	regulates	gene	0.95
NEAT1	downregulates	gene	0.95
Lc3Ylation	regulates	process	0.95
ALS-FTD	associated_with	disease	0.95
Tdp-43 Proteinopathy	involved_in	disease	0.95
Frontotemporal Lobar Degeneration	associated_with	disease	0.95
Intra-Condensate Demixing	involved_in	process	0.95
Nuclear Condensation	mediates	mechanism	0.95
STMN2	regulates	gene	0.95
Mitochondrial Dna	causes	compound	0.95
ATG4B	regulates	gene	0.95
mitochondrial DNA	associated_with	compound	0.95
RNA splicing	regulates	process	0.95
Nuclear Speckle Assembly	regulates	process	0.95
Zygotic Splicing Activation	regulates	process	0.95
Nuclear Speckle Assembly	activates	process	0.95
Ubiquitination	involved_in	process	0.95
Amyotrophic Lateral Sclerosis	risk_factor_for	disease	0.95
FTD	causes	disease	0.95
ALS	involved_in	disease	0.95
FTD	involved_in	disease	0.95
ALS	biomarker_for	disease	0.95
Amyotrophic Lateral Sclerosis	involved_in	disease	0.95
Amyotrophic Lateral Sclerosis	implicated_in	disease	0.95
HDGFL2	regulates	protein	0.93
FTLD-TDP Type C	associated_with	disease	0.93
Cryptic Splicing	mediates	process	0.92
Behavior Variant Ftd	biomarker_for	disease	0.92
UNC13A	drives	gene	0.92
FTLD-TDP C	associated_with	disease	0.92
UBIQUITIN	associated_with	protein	0.90
Motor Neuron Degeneration	causes	disease	0.90
Motor Neuron	expressed_in	cell_type	0.90
Cytoplasmic Mislocalization	associated_with	phenotype	0.90
Pathological Aggregation	associated_with	phenotype	0.90
Rna Processing	regulates	process	0.90
Motor Neuron Degeneration	contributes_to	process	0.90
ATG4B	inhibits	gene	0.90

Incoming (509)

Source	Relation	Type	Str
SOD1	associated_with	gene	1.00
FUS	associated_with	gene	1.00
ALS	associated_with	gene	1.00
AUTOPHAGY	associated_with	gene	1.00
ALZHEIMER'S DISEASE	associated_with	disease	1.00
AMYLOID	associated_with	protein	1.00
Extracellular Vesicles	transports	biomarker	0.95
Alternative Splicing	regulates	process	0.95
MAPK/ERK	phosphorylates	pathway	0.95
CASP2	modulates	protein	0.90
Tdp-43 Aggregates	associated_with	protein	0.90
Heat Shock Response	phosphorylates	process	0.90
YWHAQ	binds	protein	0.90
Pedv N Protein	degrades	protein	0.90
Up-Concentration In Stress Granules	mediates	process	0.90
Stress	activates	process	0.90
ATXN2	suppresses	gene	0.90
Ubiquitination	promotes	process	0.90
Oxidative Stress	promotes	process	0.90
h-7e846ceb	targets_gene	hypothesis	0.90
h-4fabd9ce	targets_gene	hypothesis	0.90
h-eea667a9	targets_gene	hypothesis	0.90
h-7693c291	targets_gene	hypothesis	0.90
h-47ab2be5	targets_gene	hypothesis	0.90
h-10b5bf6f	targets_gene	hypothesis	0.90
h-var-a0933e666d	targets_gene	hypothesis	0.90
Nuclear Condensation	inhibits	mechanism	0.85
CasRx	inhibits	protein	0.85
Frontotemporal Dementia	associated_with	disease	0.85
PARKINSON'S DISEASE	associated_with	gene	0.82
h-7e846ceb	targets	hypothesis	0.80
h-4fabd9ce	targets	hypothesis	0.80
h-eea667a9	targets	hypothesis	0.80
Als-Linked Mutations	modulates	biomarker	0.80
TAR	regulates	gene	0.75
NF-KB	associated_with	protein	0.74
EGFR	activates	gene	0.70
OPTN	associated_with	gene	0.70
ATG	associated_with	gene	0.70
NEURODEGENERATIVE DISEASES	associated_with	gene	0.70
NEURODEGENERATION	activates	gene	0.70
RNA	interacts_with	gene	0.70
rapamycin	targets	drug	0.70
MTOR	activates	gene	0.66
JUN	associated_with	gene	0.66
APOE	associated_with	gene	0.66
BDNF	associated_with	gene	0.66
ALS	activates	gene	0.66
AUTOPHAGY	activates	gene	0.66
C9ORF72	targets	gene	0.65

Targeting Hypotheses (4)

Hypotheses where this entity is a therapeutic target

Hypothesis	Score	Disease	Analysis
Cryptic Exon Silencing Restoration	0.462	neurodegeneration	RNA binding protein dysregulation across
Cross-Seeding Prevention Strategy	0.451	neurodegeneration	RNA binding protein dysregulation across
Glycine-Rich Domain Competitive Inhibition	0.429	neurodegeneration	TDP-43 phase separation therapeutics for
RNA-Binding Competition Therapy for TDP-43 Cross-Seeding	0.374	neurodegeneration	Protein aggregation cross-seeding across

Mentioning Analyses (4)

Scientific analyses that reference this entity

Related Papers (20)

Scientific publications cited in analyses involving this entity

Title & PMID	Authors	Journal	Year	Citations
Autophagy and ALS: mechanistic insights and therapeutic implications. [PMID:34057020]	Chua JP, De Calbiac H, Kabashi E, Barmad	Autophagy	2022	2
TDP-43 Pathology in Alzheimer's Disease. [PMID:34930382]	Meneses A, Koga S, O'Leary J, Dickson DW	Mol Neurodegener	2021	2
Protein transmission in neurodegenerative disease. [PMID:32203399]	Peng C, Trojanowski JQ, Lee VM	Nat Rev Neurol	2020	2
TDP-43 Triggers Mitochondrial DNA Release via mPTP to Activate cGAS/STING in ALS [PMID:33031745]	Yu CH, Davidson S, Harapas CR, Hilton JB	Cell	2020	2
Trehalose induces autophagy via lysosomal-mediated TFEB activation in models of [PMID:30335591]	Rusmini P, Cortese K, Crippa V, Cristofa	Autophagy	2019	2
Targets and Gene Therapy of ALS (Part 1). [PMID:40362304]	Shiryaeva O, Tolochko C, Alekseeva T, Dy	Int J Mol Sci	2025	1
The genetics of amyotrophic lateral sclerosis. [PMID:38967083]	Nijs M, Van Damme P	Curr Opin Neurol	2024	1
TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhi [PMID:39305312]	Torres P, Rico-Rios S, Ceron-Codorniu M,	Acta Neuropathol	2024	1
N protein of PEDV plays chess game with host proteins by selective autophagy. [PMID:36861818]	Zhai X, Kong N, Zhang Y, Song Y, Qin W,	Autophagy	2023	1
PIKFYVE inhibition mitigates disease in models of diverse forms of ALS. [PMID:36754049]	Hung ST, Linares GR, Chang WH, Eoh Y, Kr	Cell	2023	1
TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A. [PMID:35197628]	Brown AL, Wilkins OG, Keuss MJ, Kargbo-H	Nature	2022	1
Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP- [PMID:28405022]	Becker LA, Huang B, Bieri G, Ma R, Knowl	Nature	2017	1
Selective Silencing of TDP-43 P. G376D Mutation Reverses Key Amyotrophic Lateral [PMID:41897327]	Romano R, Ruotolo G, Perrone F, Tomasell	Biomolecules	2026	0
ALS-related proteinopathies: From TDP-43 to mitochondrial proteinopathies. [PMID:41570741]	Genin EC, Paquis-Flucklinger V	Current opinion in neurobiolog	2026	0
Chemical and Molecular Strategies in Restoring Autophagic Flux in TDP-43 Protein [PMID:41900026]	Jamerlan A, Hulme J	Molecules (Basel, Switzerland)	2026	0
Axonal transport impairment as an upstream mechanism in amyotrophic lateral scle [PMID:41890591]	Gabbay U	Frontiers in neuroscience	2026	0
Role of Alpha-Synuclein in Frontotemporal Dementia: Narrative Review. [PMID:41827903]	Bougea A	Cells	2026	0
Versatile CRISPR-Cas Tools for Gene Regulation in Zebrafish via an Enhanced Q Bi [PMID:41671402]	["Shi M", "Ge W", "Li C", "Liu B", "Deng	Advanced science (Weinheim, Ba	2026	0
Splicing the narrative: alternative TARDBP splicing and its relation to neurodeg [PMID:41837283]	Miller MR, Dykstra M, Barmada S	The Journal of clinical invest	2026	0
Multi-modal dissection of cell-type specific TDP-43 pathology in the motor corte [PMID:41803120]	Ruf WP, Kühlwein JK, Meier L, Brockmann	Nature communications	2026	0

TARDBP

Understanding Entity Pages

Summary

Wiki Pages (21)

TARDBP — TAR DNA Binding Protein 43

nimodipine-als

Frontotemporal Dementia (FTD) Mechanistic Pathway

neuroinflammation

Frontotemporal Dementia (FTD)

HNRNPA2B1 Protein

Pathway Diagram

Outgoing (729)

Incoming (509)

Targeting Hypotheses (4)

Mentioning Analyses (4)

CRISPR-based therapeutic approaches for neurodegenerative diseases

RNA binding protein dysregulation across ALS FTD and AD

TDP-43 phase separation therapeutics for ALS-FTD

Protein aggregation cross-seeding across neurodegenerative diseases

Related Papers (20)

TARDBP

Understanding Entity Pages

Summary

Wiki Pages (21)

TARDBP — TAR DNA Binding Protein 43

nimodipine-als

Frontotemporal Dementia (FTD) Mechanistic Pathway

neuroinflammation

Frontotemporal Dementia (FTD)

HNRNPA2B1 Protein

oxidative-stress-comparison

ftd-tdp-pathology

Prion vs Tau/Alpha-Synuclein Propagation: Mechanistic Comparison

Epigenetic Dysregulation in Neurodegeneration

Mitochondrial Dysfunction in Neurodegeneration

Pre-symptomatic Conversion Windows in ALS Genetic Risk Carriers

Splice-Modulating Therapies for Neurodegeneration

TDP-43 Proteinopathy

Protein Aggregation Comparison in Neurodegeneration

RNA Metabolism Dysregulation in Alzheimer's Disease

Motor Neuron Disease (MND)

Cleveland Clinic Lerner Research Institute

DDX3X Gene

Transcriptional Regulation in Neurodegeneration

Unfolded Protein Response (UPR)

Pathway Diagram

Outgoing (729)

Incoming (509)

Targeting Hypotheses (4)

Mentioning Analyses (4)

CRISPR-based therapeutic approaches for neurodegenerative diseases

RNA binding protein dysregulation across ALS FTD and AD

TDP-43 phase separation therapeutics for ALS-FTD

Protein aggregation cross-seeding across neurodegenerative diseases

Related Papers (20)