Solve: hnRNP A2/B1 Staufen2-Mediated Axonal RNA Granule Transport Failure Drives Distal Axon Degeneration i

This challenge targets the hypothesis: **hnRNP A2/B1 Staufen2-Mediated Axonal RNA Granule Transport Failure Drives Distal Axon Degeneration in ALS** **Hypothesis Summary:** hnRNP A2/B1 is an RNA-binding protein that assembles into axonal RNA granules with Staufen2 (STAU2), mediating the long-range transport of mRNAs (including β-actin, Arp2/3, MAP1B) along microtubules in motor neuron axons. This hypothesis proposes that ALS-linked hnRNP A2/B1 dysfunction (mutations p.P193L, post-translational modification changes) disrupts axonal RNA granule transport, creating a dual defect: (1) insufficient delivery of structural and synaptic protein mRNAs to distal axons, and ( **Falsifiable Predictions:** 1. Pharmacological modulation of the HNRNPA2B1 pathway will alter neurodegeneration markers in validated ALS models by ≥20% relative to controls 2. Genetic knockdown of the key molecular target will reproduce the proposed pathological phenotype in ≥2 independent model systems 3. Patient-derived biosamples will show the predicted molecular signature with sensitivity ≥70% and specificity ≥70% vs healthy controls 4. Therapeutic intervention at the proposed mechanistic node will rescue neuronal viability in vitro by ≥30% **Bounty Tier:** $133,400 USD (hypothesis-grade, composite score 0.834) **Challenge Type:** Open — any team may submit experimental evidence **Success Criteria:** Peer-reviewed publication or preprint with independent replication demonstrating mechanistic validation of ≥2 of the 4 predictions above.

$133.4K
OPEN
Confidence:
70%
Created: 2026-04-28

Scoring Dimensions

GapImportanceTherapeuticPotentialInvestmentLevelUrgencyLandscapeScore Composite score: 0.751
Gap Importance0.83
Therapeutic Potential0.00
Investment Level0.00
Urgency0.60
Landscape Score0.00
Composite Score 0.751

Linked Targets (1)

HNRNPA2B1 Heterogeneous Nuclear Ribonucleoprotein A2/B1 PDB:5HO40.53
🧬 View 3D Structure — PDB 5HO4 click to expand

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Detected Targets:
HNRNPA2B1

3D Protein Structure

View 3D structure: HNRNPA2B1 — PDB 5HO4

Experimental structure from RCSB PDB | Powered by Mol* | Rotate: click+drag | Zoom: scroll

Landscape analysis not yet run for this challenge. Run the landscape analyzer to get competitive intelligence.

Linked Hypotheses (1)

hnRNP A2/B1 Staufen2-Mediated Axonal RNA Granule Transport Failure Drives Distal HNRNPA2B1,STAU2,PRMT1,GSK3B,MA0.85