🧪
hypothesis

VPS35 Retromer Restoration to Rescue Endosomal Protein Trafficking

Hypothesis

VPS35 Retromer Restoration to Rescue Endosomal Protein Trafficking

VPS35 Retromer Restoration to Rescue Endosomal Protein Trafficking.
🧬 VPS35 (VPS26/VPS29/VPS35 complex)🩺 proteomics🎯 Composite 52%💱 $0.53▲4.5%proposed
EvidencePending (0%)📖 0 cit🗣 1 debates 5 support 6 oppose
⚠ Missing Evidence⚠ Thin Description Senate Quality Gates →
Mechanistic 0.65 (15%) Evidence 0.53 (15%) Novelty 0.55 (12%) Feasibility 0.55 (12%) Impact 0.60 (12%) Druggability 0.55 (10%) Safety 0.50 (8%) Competition 0.45 (6%) Data Avail. 0.50 (5%) Reproducible 0.55 (5%) KG Connect 0.50 (8%) 0.525 composite
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arXiv PreprintNeurIPSNature MethodsPLOS ONE
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Composite52%

🧪 Overview

VPS35 Retromer Restoration to Rescue Endosomal Protein Trafficking

🧬 Mechanism

🧬 Curated Mechanism Pathway

Curated pathway from expert analysis

flowchart TD
    A["VPS35-VPS26-VPS29<br/>Retromer Core Trimer"]
    B["Endosomal Cargo Recognition<br/>CI-MPR/ATG9/SorLA Retrieval"]
    C["Retrograde Trafficking<br/>Endosome-to-TGN"]
    D["WASH Complex Recruitment<br/>Actin Branching on Endosome"]
    E["Cathepsin D Maturation<br/>Lysosomal Hydrolase Sorted"]
    F["VPS35 D620N Mutation<br/>Parkinson's PARK17"]
    G["Lysosomal Dysfunction<br/>Alpha-Synuclein Accumulation"]
    A --> B
    B --> C
    C --> D
    C --> E
    F -.->|"impairs"| A
    F --> G
    style A fill:#1a237e,stroke:#4fc3f7,color:#4fc3f7
    style E fill:#1b5e20,stroke:#81c784,color:#81c784
    style F fill:#b71c1c,stroke:#ef9a9a,color:#ef9a9a
    style G fill:#b71c1c,stroke:#ef9a9a,color:#ef9a9a

⚖️ Evidence

⚖️ Evidence Matrix5 supports6 contradicts
Supports
VPS35 mutations cause autosomal-dominant Parkinson's disease with synaptic dysfunction
PMID:21725305
Supports
Retromer protein levels are reduced in AD hippocampus and correlate with cognitive decline
PMID:25898100
Supports
Retromer dysfunction causes APP mislocalization to endosomes, increasing Aβ production
PMID:23792953
Supports
R55 compound rescues VPS35 mutations and restores retromer function in cellular models
PMID:23499328
Supports
Retromer mediates retrieval of synaptic receptors (APP, Vps10, SorLA) from degradative pathway
PMID:27457933
Contradicts
VPS35 mutations cause Parkinson's, not Alzheimer's - mechanistic disconnect
PMID:21725305
Contradicts
VPS35 overexpression in mouse models causes dopamine neuron degeneration
PMID:30270026
Contradicts
Retromer enhancement increases Aβ production in some cellular models by redirecting APP to amyloidogenic compartments
PMID:27457933
Contradicts
R55 compound validation limited to HeLa cells and yeast; no human neuron data
PMID:23499328
Contradicts
Retromer affects thousands of cargo including Wntless, glutamate receptors, transferrin receptor
PMID:25898100
Contradicts
Correlation between VPS35 levels and cognitive decline may be secondary to neurodegeneration
PMID:25898100
📖 Linked Papers

No linked papers recorded for this hypothesis yet.

🏥 Translation

🧬 3D Protein Structure — VPS35

No curated PDB or AlphaFold mapping for VPS35 yet. Search RCSB →

🧠 GTEx v10 Brain ExpressionJSON

Median TPM across 13 brain regions for VPS35 (VPS26/VPS29/VPS35 complex) from GTEx v10.

Cerebellar Hemisphere34.4 Frontal Cortex BA933.4 Cerebellum26.2 Hypothalamus25.3 Cortex22.4 Spinal cord cervical c-120.6 Anterior cingulate cortex BA2420.6 Nucleus accumbens basal ganglia19.9 Caudate basal ganglia18.0 Substantia nigra16.9 Hippocampus14.7 Amygdala13.7 Putamen basal ganglia13.4median TPM (GTEx v10)

💉 Clinical Trials

No clinical trials data linked to this hypothesis yet.

No curated ClinVar variants loaded for this hypothesis.

Run scripts/backfill_clinvar_variants.py to fetch P/LP/VUS variants.

🔍 Search ClinVar for VPS35 (VPS26 →

No DepMap CRISPR Chronos data found for VPS35 (VPS26.

Run python3 scripts/backfill_hypothesis_depmap.py to populate.

💰 Estimated Development
Cost
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📊 Market Indicators

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💾 Resource Usage

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🔮 Predictions

🔎 Predictions vs Observations2 predictions · 0 with recorded observations
PredictionPredictedObservedStatusConf
IF VPS35 protein levels are restored to ≥80% of wild-type levels in VPS35-knockdown SH-SY5Y neuronal cells via CRISPR-activation, THEN the surface expression of the retromer cargo protein SorLA will iSorLA surface expression will increase by ≥2-fold, quantified by cell-surface biotinylation assay and flow cytometry.— no observation —pending0.60
IF VPS35 expression is restored via viral transduction (AAV-VPS35) in VPS35-deficient patient-derived fibroblasts, THEN the rate of transferrin receptor recycling will increase by at least 40% compareTransferrin receptor recycling rate will increase by ≥40% relative to baseline deficiency, measured by live-cell imaging of fluorescent transferrin.— no observation —pending0.65
🔮 Falsifiable Predictions (2)
pendingconf 65%
IF VPS35 expression is restored via viral transduction (AAV-VPS35) in VPS35-deficient patient-derived fibroblasts, THEN the rate of transferrin receptor recycling will increase by at least 40% compared to non-transduced VPS35-deficient cells within 72 hours post-transduction.
Predicted outcome: Transferrin receptor recycling rate will increase by ≥40% relative to baseline deficiency, measured by live-cell imaging of fluorescent transferrin.
Falsification: Transferrin receptor recycling rate remains below 20% of wild-type levels, or endosomal cargo mislocalization persists (≥80% of cells showing cytoplasmic aggregation of retromer cargo proteins CI-M6PR
pendingconf 60%
IF VPS35 protein levels are restored to ≥80% of wild-type levels in VPS35-knockdown SH-SY5Y neuronal cells via CRISPR-activation, THEN the surface expression of the retromer cargo protein SorLA will increase by at least 2-fold compared to non-activated knockdown cells within 48 hours.
Predicted outcome: SorLA surface expression will increase by ≥2-fold, quantified by cell-surface biotinylation assay and flow cytometry.
Falsification: SorLA surface expression shows no statistically significant increase (p>0.05, n=4 replicates), or total cellular SorLA levels remain ≥50% below wild-type controls indicating trafficking block persists
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