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R653C

Category 4 — Stable Fold, Function DisruptedConflictingLumenal · predictedσ-1 candidateEditorial
ArginineCysteine at position 653 · C-terminal lumenal domain (653-869) · WFS1 (Wolframin)

Arginine → Cysteine at position 653 in lumenal domain. ClinVar Conflicting. AlphaMissense 0.476 (below threshold), ΔΔG +0.22. pLDDT 61 borderline.

Interactive 3D Structure

Wild-type reference
Wild-type R653 — hydrogen bond to Y650
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DynaMut2 mutant · R653C
Mutant C653 — hydrogen bond contact to F649 lost
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Bond changes · DynaMut2 interaction analysis

1 lost1 gained9 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondF649F649Preserved
Hydrogen bondY650Y650Preserved
Hydrogen bondG656G656Preserved
Polar contactF649F649Preserved
Polar contactY650Y650Preserved
Polar contactV651V651Preserved
Polar contactG656G656Preserved
Van der WaalsY650Gained
Van der WaalsV651V651Preserved
Van der WaalsE655E655Preserved
HydrophobicF649Lost

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
0.22kcal/mol
Stabilising — mild
AlphaMissense
0.476
Amb
AlphaFold pLDDT
61
model confidence
Schema
Cat 4
Category 4 — Stable Fold, Function Disrupted
ΔΔG confidence: Reduced confidence · Lumenal
  • pLDDT 61.31 below 70

The underlying AlphaFold model has pLDDT below 70 at this position, so the structure the stability calculation was run on is itself uncertain.

Do not rank this value against a variant from a different region on ΔΔG alone.

Clinical Evidence

ClinVar classificationConflicting classifications of pathogenicity
Review statuscriteria provided, conflicting classifications
Associated conditionsWFS1-Related Spectrum Disorders
InheritanceWFS1 spectrum.
Population frequency (gnomAD v4)Low frequency · AF 0.028%
cDNA changec.1957C>T
ClinVar accessionVCV000178597
Last evaluated2026/01/24 00:00

Observed in the general population.

Classified for1★ unverified submission

ClinVar classifies this variant as Conflicting classifications of pathogenicity for WFS1-related spectrum (unresolved mode) (dominant or recessive); Nonsyndromic hearing loss (DFNA6/14/38) (autosomal dominant, OMIM 600965); WFS1-related diabetes (autosomal dominant); Wolfram syndrome (classic) (autosomal recessive, OMIM 222300); Cataract 41 (autosomal dominant, OMIM 116400); Wolfram-like syndrome (autosomal dominant, OMIM 614296); Retinal dystrophy (inheritance not specified). ClinVar records this under a WFS1-spectrum label that spans both disease families — recessive Wolfram syndrome (biallelic) and the dominant WFS1-related disorders. The label does not resolve which applies, and it is not a severity statement.

  • WFS1-related spectrum (unresolved mode)dominant or recessivesubmitted as: WFS1-Related Spectrum Disorders
  • Nonsyndromic hearing loss (DFNA6/14/38)autosomal dominant · OMIM 600965submitted as: Autosomal dominant nonsyndromic hearing loss 6; Autosomal dominant nonsyndromic hearing loss 6
  • WFS1-related diabetesautosomal dominantsubmitted as: Type 2 diabetes mellitus
  • Wolfram syndrome (classic)autosomal recessive · OMIM 222300submitted as: Wolfram syndrome 1
  • Cataract 41autosomal dominant · OMIM 116400submitted as: Cataract 41
  • Wolfram-like syndromeautosomal dominant · OMIM 614296submitted as: Wolfram-like syndrome
  • Retinal dystrophyinheritance not specifiedsubmitted as: Retinal dystrophy
  • Submitters conflict on this classification (1★). Treat the conditions above as submitted, not as documented phenotypes.
  • Phenotype scope is interpretation of submitted records. The computed values on this card (AlphaMissense, DynaMut2 ΔΔG, pLDDT) are measurements of the model, not of a patient.
  • Presentation cannot be generalised from neighbouring variants: one nucleotide over can present very differently. Structural similarity supports a mechanistic hypothesis, never a phenotype claim.

Review status: 1★ conflicting classifications. ClinVar "conditions" is the union of all submissions; 0–1★ entries are submitted conditions, not documented phenotypes.

Population frequency
Global (all ancestries)
AF 0.028% · 457 / 1,613,828 alleles
Homozygotes
2
Highest-frequency population
Ashkenazi Jewish · AF 0.557%

Highest in Ashkenazi Jewish: AF 0.557% (165 of 29,608 alleles), 19.7x the global figure. The global AF describes the general population, not the at-risk group.

2 homozygotes reported in gnomAD v4 (1 Ashkenazi Jewish; 1 Admixed American). gnomAD excludes individuals with severe pediatric disease, so homozygotes in this dataset argue against a fully penetrant severe recessive phenotype and belong in the clinical picture. This is interpretive signal, not a classification.

Ancestry groupAllele freq.AC / ANHom.Carrier est.
Ashkenazi Jewish0.557%165 / 29,6081~1 in 90
Admixed American0.183%110 / 60,0321~1 in 270
East Asian0.085%38 / 44,8740~1 in 590
Remaining individuals0.062%39 / 62,5060~1 in 800
South Asian0.016%15 / 91,0900~1 in 3040
European (non-Finnish)0.0072%85 / 1,180,0240~1 in 6940
African / African American0.0053%4 / 75,0580~1 in 9380
Finnish · under-sampled0.0016%1 / 63,6640

Source: gnomAD r4, retrieved 2026-08-21. Values are allele frequencies. The carrier estimate is derived from the allele frequency (Hardy-Weinberg, ~2·AF) and is an approximation, not a published figure — where a published carrier frequency exists it outranks this column and is cited on the card.

Structural Context

Position 653 at the lumenal domain start. Neighbors: SER654 (2.5 Å), TYR652 (2.5 Å), TYR650 (3.6 Å — Y650H/Y650D/Y650C region!), PHE649 (3.8 Å — Y650 cluster).

R653C sits adjacent to the Y650 aromatic cluster (multiple Atlas variants at Y650). The introduced thiol could engage in aberrant disulfide chemistry. AM 0.476 below threshold but multi-phenotype + ClinVar Pathogenic raise concern.

Amino-acid chemistry
Arginine (R) → Cysteine (C) — charge loss + thiol introduction.
Position in the protein
C-terminal lumenal domain · position 653 at the start of the lumenal domain (pLDDT 61 borderline).

Druggability Assessment

Category 4 — Stable Fold, Function Disrupted (AM below threshold, pLDDT borderline). ΔΔG +0.22. AlphaMissense 0.476 below threshold.

Mechanism: charge loss + thiol near Y650 cluster. Therapeutic: same Y650 microregion. Wet-lab validation recommended.

Why this matters

R653C joins the AM-under-call class. Adjacency to Y650 cluster makes it of interest despite the borderline signals.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the R653C 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 R653C PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Topological domain653869 · Lumenal
Natural variant653653 · in DFNA6; uncertain significance; also found in a patient with type 2 diabetes; uncertain significance; dbSNP:rs201064551