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P292S

Category 3/4 — Most DruggableLikely pathogenicCytoplasmic · predictedEditorial
ProlineSerine at position 292 · N-terminal cytoplasmic domain (87-313) · WFS1 (Wolframin)

Proline → Serine at position 292 in wolframin's N-terminal cytoplasmic domain. ClinVar Likely pathogenic for classical Wolfram syndrome 1. AlphaMissense 0.957, DynaMut2 ΔΔG -0.74 kcal/mol (destabilising). Critically, pLDDT at this position is 54 — just above the Category 5 IDR threshold but in a low-confidence region.

Interactive 3D Structure

Wild-type reference
Wild-type P292 — hydrogen bond to I296
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DynaMut2 mutant · P292S
Mutant S292 — van der waals contact to H294 lost
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Bond changes · DynaMut2 interaction analysis

1 lost0 gained4 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondI296I296Preserved
Polar contactH294H294Preserved
Polar contactA295A295Preserved
Polar contactI296I296Preserved
Van der WaalsH294Lost

Lost / gained / preserved interatomic contacts at the variant residue, from the DynaMut2 (Arpeggio) interaction analysis of the wild-type and energy-minimized mutant structures.

Computational Predictions

DynaMut2 ΔΔG
-0.74kcal/mol
Destabilising — mild
AlphaMissense
0.957
LPath
AlphaFold pLDDT
54
model confidence
Schema
Cat 3/4
Category 3/4 — Most Druggable

Clinical Evidence

ClinVar classificationLikely pathogenic
Review statuscriteria provided, single submitter
Associated conditionsWolfram syndrome 1
InheritanceAutosomal recessive Wolfram syndrome 1 phenotype documented in ClinVar.
Population frequency (gnomAD v4)Ultra-rare · AF 0.0011%
cDNA changec.874C>T
ClinVar accessionVCV003377398
Last evaluated2022/03/31 00:00

Observed at very low frequency in gnomAD.

Structural Context

Position 292 sits in wolframin's N-terminal cytoplasmic domain. The AlphaFold model places P292 within 5 Å of LEU293 (2.5 Å), TYR291 (2.5 Å), VAL288 (3.6 Å), HIS294 (4.4 Å), and ALA295 (4.7 Å). The local environment is mixed hydrophobic-polar.

The wild-type proline at 292 likely plays a deliberate structural role — proline residues in cytoplasmic domains often define loop boundaries or contribute backbone constraints that orient the domain for partner binding. Replacing it with serine removes that backbone constraint and adds a hydroxyl group with H-bond capacity.

The |ΔΔG| of 0.74 kcal/mol is interpretively complicated by the pLDDT of 54. DynaMut2 assumes a meaningful input structure; at pLDDT 54 the local AlphaFold prediction has lower confidence than the typical Atlas variant. The ΔΔG should be read as a directional indicator rather than a quantitative claim. AlphaMissense's 0.957 score is independently supportive — the substitution is pathogenic even if the structural mechanism is partially obscured by model uncertainty.

ClinVar Likely Pathogenic for classical Wolfram syndrome 1 confirms the variant's clinical relevance.

Amino-acid chemistry
Proline (P) → Serine (S) — a rigid, helix-breaking residue replaced by a small polar hydroxyl-bearing residue. The substitution removes the proline's backbone constraint and introduces hydrogen-bonding capacity.
Position in the protein
N-terminal cytoplasmic domain · position 292 sits in a region with relatively low AlphaFold confidence (pLDDT 54), just above the Cat 5 IDR threshold of 50. Interpret structural predictions here with caution.

Druggability Assessment

Category 3/4 — Most Druggable (with caveat). |ΔΔG| = 0.74 kcal/mol below the fold-integrity threshold. AlphaMissense 0.957 confirms pathogenic signal. However, pLDDT of 54 means the structural details deserve experimental validation before drug design proceeds.

The mechanism is removal of a deliberate proline backbone constraint plus introduction of a polar hydroxyl. Therapeutic strategy: a small molecule that stabilizes the wild-type backbone geometry around the position 292 loop region. Pharmacological chaperone screening is the safer initial approach given the structural confidence caveat.

Why this matters

P292S sits in a borderline-confidence region of the AlphaFold model. The Atlas captures this with the pLDDT score; pre-atlas drug discovery would have either treated the variant at full structural confidence (overconfident) or ignored it entirely (under-utilizing the AlphaMissense signal). The dual-metric framing makes the appropriate caution visible — proceed with chaperone screening, validate experimentally, then proceed to docking once the structural geometry is confirmed.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the P292S PDF below and upload it to Wolfram Intelligence to generate therapeutic-strategy proposals — guanidinium mimetics, sigma-1 agonist docking, NAC thiol-capping. NAC is already on the bench-testing list.

Download P292S PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Region1321 · Interaction with ATP6V1A
Natural variant292292 · in WFS1; dbSNP:rs746923441