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C847Y

Category 3/4 — Most DruggableConflictingLumenal · predictedσ-1 candidateEditorial
CysteineTyrosine at position 847 · C-terminal lumenal domain (653-869) · WFS1 (Wolframin)

Cysteine → Tyrosine at position 847 in wolframin's C-terminal lumenal domain. ClinVar Conflicting classifications including Wolfram syndrome 1 and autistic behavior. AlphaMissense 0.994 (near-maximum), DynaMut2 ΔΔG -0.92 kcal/mol (destabilising). Cysteine-removal with potential disulfide loss to C850.

Interactive 3D Structure

Wild-type reference
Wild-type C847 — hydrogen bond to R859
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DynaMut2 mutant · C847Y
Mutant Y847 — hydrogen bond contact to C850 lost
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Bond changes · DynaMut2 interaction analysis

1 lost6 gained6 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondV813Gained
Hydrogen bondC850C850Preserved
Hydrogen bondR859R859Preserved
Polar contactS816Gained
Polar contactC850C850Preserved
Polar contactR859R859Preserved
Van der WaalsC850Gained
Van der WaalsR859Gained
HydrophobicV813Gained
HydrophobicL817L817Preserved
HydrophobicI823Lost
HydrophobicC850C850Preserved
HydrophobicR859Gained

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
-0.92kcal/mol
Destabilising — mild
AlphaMissense
0.994
LPath
AlphaFold pLDDT
86
model confidence
Schema
Cat 3/4
Category 3/4 — Most Druggable

Clinical Evidence

ClinVar classificationConflicting classifications of pathogenicity
Review statuscriteria provided, conflicting classifications
Associated conditionsWolfram syndrome 1; Autistic behavior
InheritanceConflicting classifications including Wolfram syndrome 1.
Population frequency (gnomAD v4)Ultra-rare · AF 0.00081%
cDNA changec.2540G>A
ClinVar accessionVCV000437421
Last evaluated2024/07/05 00:00

Observed at very low frequency in gnomAD.

Structural Context

Position 847 sits in wolframin's C-terminal lumenal domain. The AlphaFold model places C847 within 5 Å of SER846 (2.4 Å), LEU848 (2.5 Å), LEU822 (3.8 Å — long-range), ILE823 (4.3 Å), and CYS850 (4.4 Å — another cysteine!). The C847-C850 distance of 4.4 Å is consistent with a possible structural disulfide bond.

Replacing C847 with tyrosine eliminates this potential disulfide and introduces aromatic volume. The C850 partner residue (partner of C850Y Atlas card) loses its disulfide partner if one existed. The |ΔΔG| of 0.92 reflects substantial fold cost.

AlphaMissense 0.994 confirms severe functional consequence. The C847-C850 microregion has variants at both cysteines (C847Y and C850Y), suggesting both cysteines are structurally important and disulfide-related.

Amino-acid chemistry
Cysteine (C) → Tyrosine (Y) — thiol-bearing residue replaced by aromatic phenol. Loss of disulfide potential; aromatic introduction.
Position in the protein
C-terminal lumenal domain · position 847 in the ER lumen (pLDDT 86).

Druggability Assessment

Category 3/4 — Most Druggable. |ΔΔG| = 0.92 — fold survives. AlphaMissense 0.994 confirms severe functional consequence.

Mechanism is potential C847-C850 disulfide loss plus aromatic volume introduction. Therapeutic strategy: site-directed at the C847-C850 microregion.

Why this matters

C847Y + C850Y are sister variants at adjacent cysteines, possibly a structural disulfide pair. The Atlas now identifies THREE possible disulfide pairs in the lumenal domain: C673-C690, C733-C765, C847-C850. The lumenal fold appears to rely on multiple disulfides for structural integrity.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the C847Y 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 C847Y PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Topological domain653869 · Lumenal