cGAMP biomarker analysis in ALS patient spinal cord samples

Exploratory Score: 0.800 Price: $0.50 Amyotrophic Lateral Sclerosis Human ALS patient spinal cord tissue Status: proposed

What This Experiment Tests

Exploratory experiment designed to discover new patterns targeting TARDBP in Human ALS patient spinal cord tissue. Primary outcome: Elevated cGAMP levels as biomarker

Description

Post-mortem analysis of spinal cord samples from ALS" class="entity-link entity-disease" title="disease: ALS">ALS patients to measure levels of cyclic GMP-AMP (cGAMP), the specific metabolite produced by cGAS enzyme activation. This study aimed to validate the clinical relevance of the cGAS/STING pathway in human ALS pathology by demonstrating elevated cGAMP levels as a potential biomarker of mitochondrial DNA release and cGAS/STING activation. The analysis provided crucial translational evidence connecting the mechanistic findings from cellular and animal models to human disease pathology.

TARGET GENE

TARDBP (Score: 0.510)

MODEL SYSTEM

Human ALS patient spinal cord tissue

ESTIMATED COST

TIMELINE

0 months

PATHWAY

cGAS/STING pathway

SOURCE

extracted_from_pmid_33031745

PRIMARY OUTCOME

Elevated cGAMP levels as biomarker

Scoring Dimensions

📖 Wiki Pages

TARDBP/TDP-43 Proteinprotein TARDBP Protein (TDP-43)protein TARDBP — TAR DNA Binding Protein 43gene TARDBP Mutations in Amyotrophic Lateral Sclerosisdisease STING Inhibitor Therapy in Neurodegenerationtherapeutic DNA Repair Therapy for Neurodegenerationtherapeutic DNA Damage Repair Therapy for Neurodegenerationtherapeutic ALS Treatment Strategiestherapeutic ALS Treatment Overviewtherapeutic ALS Therapeuticstherapeutic ALS Therapeutic Landscape — Programs by Phase and disease ALS Cure Roadmaptherapeutic ALS Communication Brain-Computer Interfacestechnology STING Proteinprotein STING Proteinredirect

Protocol

Post-mortem spinal cord tissue collection, cGAMP quantification assays, comparison between ALS" class="entity-link entity-disease" title="disease: ALS">ALS patients and controls, correlation with TDP-43 pathology

Expected Outcomes

ALS" class="entity-link entity-disease" title="disease: ALS">ALS patients should show significantly elevated cGAMP levels compared to controls, indicating active cGAS/STING signaling

Success Criteria

Statistically significant elevation of cGAMP levels in ALS" class="entity-link entity-disease" title="disease: ALS">ALS patient samples versus controls

Related Hypotheses (4)

Cryptic Exon Silencing Restoration0.462

Cross-Seeding Prevention Strategy0.451

Glycine-Rich Domain Competitive Inhibition0.429

RNA-Binding Competition Therapy for TDP-43 Cross-Seeding0.374

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