mechanism 2,601 words KG: ent-gene-9a7e7e49 2026-03-26
kind:section:mechanismsevidence:strongmechanismsection:mechanismsstate:publishedtopic:causal-chaintopic:vcptopic:tdp-43topic:alstopic:ftd
Contents

VCP→TDP-43→ALS/FTD Causal Chain

🔬 Protein Info
Gene SymbolVCP
Chromosome9p13.3
Protein FamilyAAA+ ATPase family
Protein TypeEnzyme
Target ClassEnzyme
FunctionAAA+ ATPase, protein quality control
Mechanism of ActionSmall molecule inhibitors of VCP ATPase activity affecting protein quality control
Primary ExpressionUbiquitous - Brain, Muscle, Spinal cord
Subcellular LocalizationCytoplasm, nucleus, ER, mitochondria
DruggabilityMedium (0.50)
Clinical StagePhase I
Molecular Weight97 kDa
Amino Acids806 aa
Exons17
PathwaysAkt, Autophagy, Er Stress, Mitophagy, Ubiquitin-Proteasome
UniProt IDP55072
Ensembl IDENSG00000165280
GeneCardsVCP
Human Protein AtlasVCP
Associated DiseasesALS, AMI, AUTOPHAGY, Alzheimer, Alzheimer's Disease
Known Drugs/CompoundsRAPAMYCIN, rapamycin
InteractionsTBK1, CHMP2B, OVERVIEW, VALOSIN, JUN, RANK
SciDEX TargetView Target Profile (4 clinical trials)
SciDEX HypothesesVCP-Mediated Autophagy Enhancement
VCP-Mediated Autophagy Enhancement
KG Connections957 knowledge graph edges
DatabasesGeneCardsNCBI GeneHPASTRING

Knowledge Graph

Related Hypotheses (10)

Cryptic Exon Silencing Restoration
Score: 0.46
Axonal RNA Transport Reconstitution
Score: 0.45
Cross-Seeding Prevention Strategy
Score: 0.45
Glycine-Rich Domain Competitive Inhibition
Score: 0.43
VCP-Mediated Autophagy Enhancement
Score: 0.41

Related Analyses (30)

TDP-43 phase separation therapeutics for ALS-FTD
neurodegeneration · archived
RNA binding protein dysregulation across ALS FTD and AD
neurodegeneration · archived
CRISPR-based therapeutic approaches for neurodegenerative di
neurodegeneration · archived
Microglial subtypes in neurodegeneration — friend vs foe
neuroscience · failed
CRISPR-based therapeutic approaches for neurodegenerative di
neurodegeneration · completed

Related Experiments (30)

Proposed experiment from debate on TDP-43 undergoes liquid-l
falsification · proposed · Score: 0.40
Autophagy Enhancement Drug Screening for Neurodegeneration
clinical · proposed · Score: 0.40
Mechanism: C9orf72 Hexanucleotide Repeat Expansion in ALS/FT
validation · proposed · Score: 0.40
C9orf72 Phenotype Divergence: ALS vs FTD Mechanism Study
clinical · proposed · Score: 0.40
Frontal and Temporal Lobe Selective Vulnerability in FTD — M
validation · proposed · Score: 0.40

Knowledge Graph (2 edges)

ent-gene-9a7e7e49 data_in benchmark_ot_ad_answer_key:VCP
benchmark_ot_ad_answer_key:VCP data_in ent-gene-9a7e7e49