entity

cryptic_exon_silencing

Entity Detail — Knowledge Graph Node

Understanding Entity Pages

This page aggregates everything SciDEX knows about cryptic_exon_silencing: 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.

2Connections
1Hypotheses
1Analyses
1Outgoing
1Incoming
10Experiments
2Debates

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Outgoing (1)

TargetRelationTypeStr
ALSdysregulated_indisease0.80

Incoming (1)

SourceRelationTypeStr
TDP-43regulatesprotein0.80

Targeting Hypotheses (1)

Hypotheses where this entity is a therapeutic target

HypothesisScoreDiseaseAnalysis
Cryptic Exon Silencing Restoration 0.531 neurodegeneration RNA binding protein dysregulation across

Mentioning Analyses (1)

Scientific analyses that reference this entity

RNA binding protein dysregulation across ALS FTD and AD

neurodegeneration | 2026-04-01 | 7 hypotheses Top: 0.720

Experiments (10)

Experimental studies targeting or related to this entity

ExperimentTypeDiseaseScoreFeasibilityModelStatusEst. Cost
TDP-43 mutant mouse model cGAS/STING pathway analysis validation amyotrophic lateral sclerosis 0.900 0.00 TDP-43 mutant mice proposed N/A
TDP-43 mitochondrial invasion and DNA release via mPTP exploratory amyotrophic lateral sclerosis 0.900 0.00 cultured cells proposed N/A
TDP-43 mitochondrial invasion and mtDNA release in iPSC motor neurons exploratory Amyotrophic Lateral Sclerosis 0.900 0.00 iPSC-derived motor neurons proposed N/A
cGAS/STING pathway validation in TDP-43 mutant mice validation Amyotrophic Lateral Sclerosis 0.850 0.00 TDP-43 mutant mice proposed N/A
cGAMP biomarker analysis in ALS patient spinal cord samples exploratory Amyotrophic Lateral Sclerosis 0.800 0.00 Human ALS patient spinal cord proposed N/A
TDP-43 pathology prevalence and distribution in AD cases exploratory Alzheimer's disease 0.800 0.00 human postmortem brain tissue proposed N/A
Cognitive impact of TDP-43 pathology in AD patients clinical Alzheimer's disease 0.700 0.00 human patients completed N/A
s:** - Temporal analysis showing mitochondrial defects precede other p falsification Neurodegeneration 0.400 0.50 cell_line proposed $80,000
Proposed experiment from debate on TDP-43 undergoes liquid-liquid phas falsification Neurodegeneration 0.400 0.50 cell_line proposed $80,000
s:** - Single-cell RNA-seq to measure editing efficiency across differ falsification ALS 0.400 0.50 cell_line proposed $150,000

Related Papers (20)

Scientific publications cited in analyses involving this entity

Title & PMIDAuthorsJournalYearCitations
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
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
A neurotoxic cryptic peptide arising from TDP-43-dependent cryptic splicing of P [PMID:41720774] ["Yang M", "Wang Q", "Yan R", "Kang D", Nature communications 2026 0
The Genetics of TDP-43 Type C Neurodegeneration: A Whole-Genome Sequencing Study [PMID:41883703] Nassan M, Ayala I, Sloan J, Bonfitto A, Neurology. Genetics 2026 0
Excitotoxicity in amyotrophic lateral sclerosis: a key pathogenic mechanism. [PMID:41890274] Silva-Hucha S, Hernández RG, Baena-López Brain communications 2026 0
PPAR-Delta Agonist Therapies Did Not Rescue Hallmark Disease Phenotypes in Two S [PMID:41751955] ["Luong D", "Niu C", "Kim E", "Tanji N", International journal of molec 2026 0
Transcriptomic signature of frontotemporal lobar degeneration with TDP-43 type C [PMID:41789476] ["Rajicic A", "Mol M", "Melhem S", "Kisi Brain : a journal of neurology 2026 0
Antisense oligonucleotide targeting TARDBP-EGFR splicing axis inhibits progressi [PMID:41540015] Ni N, Wang M, Yuan Z, Zhang L, Cai J, Du International journal of oral 2026 0
TDP-43 impairs glycolysis by sequestering hexokinase 1 in amyotrophic lateral sc [PMID:41838122] Barone C, Wang R, Cooke S, Ng HP, Ferrei Acta neuropathologica 2026 0

Debates (2)

Multi-agent debates referencing this entity

RNA binding protein dysregulation across ALS FTD and AD

closed · Rounds: 4 · Score: 0.72 · 2026-04-12

RNA binding protein dysregulation across ALS FTD and AD

closed · Rounds: 4 · Score: 0.88 · 2026-04-06

Related Research

Hypotheses and analyses mentioning cryptic_exon_silencing in their description or question text

No additional research found