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R703C

Category 4 — Stable Fold, Function DisruptedConflictingLumenal · predictedσ-1 candidateEditorial
ArginineCysteine at position 703 · C-terminal lumenal domain (653-869) · WFS1 (Wolframin)

Arginine → Cysteine at position 703 in lumenal domain. ClinVar Conflicting including Cataract 41 + Wolfram + DFNA6. AlphaMissense 0.471 (below threshold), ΔΔG +0.02. AM under-call with multi-phenotype.

Interactive 3D Structure

Wild-type reference
Wild-type R703 — ionic bond to E795
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DynaMut2 mutant · R703C
Mutant C703 — ionic bond to E795 lost (6 contacts lost)
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Bond changes · DynaMut2 interaction analysis

6 lost1 gained6 preserved
Interaction typeWild-type partnerMutant partnerStatus
Ionic bondE795Lost
Hydrogen bondV779Lost
Hydrogen bondG780G780Preserved
Hydrogen bondE795Lost
Hydrogen bondS821S821Preserved
Polar contactK705K705Preserved
Polar contactG780G780Preserved
Polar contactE795Lost
Polar contactS821S821Preserved
CarbonylS821S821Preserved
Van der WaalsE795Lost
Van der WaalsS821Gained
HydrophobicL822Lost

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
0.02kcal/mol
Stabilising — mild
AlphaMissense
0.471
Amb
AlphaFold pLDDT
89
model confidence
Schema
Cat 4
Category 4 — Stable Fold, Function Disrupted
ΔΔG confidence: Standard confidence · Lumenal

This variant is in a water-facing region, which is the regime the stability predictor was built and benchmarked for. Ordinary predictor error still applies.

Clinical Evidence

ClinVar classificationConflicting classifications of pathogenicity
Review statuscriteria provided, conflicting classifications
Associated conditionsCataract 41; Wolfram syndrome 1; Autosomal dominant nonsyndromic hearing loss 6 (DFNA6)
InheritanceMulti-phenotype AD.
Population frequency (gnomAD v4)Ultra-rare · AF 0.0061%
cDNA changec.2107C>T
ClinVar accessionVCV001299576
Last evaluated2025/12/15 00:00

Observed at very low frequency in gnomAD.

Classified for1★ unverified submission

ClinVar classifies this variant as Conflicting classifications of pathogenicity for Cataract 41 (autosomal dominant, OMIM 116400); Wolfram syndrome (classic) (autosomal recessive, OMIM 222300); Nonsyndromic hearing loss (DFNA6/14/38) (autosomal dominant, OMIM 600965); WFS1-related diabetes (autosomal dominant); Wolfram-like syndrome (autosomal dominant, OMIM 614296). These are two clinically distinct disease families: classic Wolfram syndrome requires two affected copies, while the dominant WFS1-related disorders do not. One label covers both, and it does not tell you which applies to a given person.

  • Cataract 41autosomal dominant · OMIM 116400submitted as: Cataract 41
  • Wolfram syndrome (classic)autosomal recessive · OMIM 222300submitted as: Wolfram syndrome 1; Wolfram syndrome 1
  • Nonsyndromic hearing loss (DFNA6/14/38)autosomal dominant · OMIM 600965submitted as: Autosomal dominant nonsyndromic hearing loss 6
  • WFS1-related diabetesautosomal dominantsubmitted as: Type 2 diabetes mellitus
  • Wolfram-like syndromeautosomal dominant · OMIM 614296submitted as: Wolfram-like syndrome
  • 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 0.0061% · 98 / 1,612,886 alleles
Homozygotes
0
Highest-frequency population
South Asian · AF 0.021%

Highest in South Asian: AF 0.021% (19 of 91,080 alleles), 3.4x the global figure. The global AF describes the general population, not the at-risk group.

No homozygotes reported in gnomAD v4. For a recessive allele this is expected and is not, on its own, evidence either way.

Ancestry groupAllele freq.AC / ANHom.Carrier est.
South Asian0.021%19 / 91,0800~1 in 2400
Middle Eastern · under-sampled0.016%1 / 6,0620
East Asian0.011%5 / 44,8700~1 in 4490
European (non-Finnish)0.0059%70 / 1,179,8460~1 in 8430
Remaining individuals0.0032%2 / 62,4920~1 in 15620
Admixed American · under-sampled0.0017%1 / 60,0180

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 703 in lumenal domain near R708 region. Neighbors: PHE704 (2.4 Å — partner of V707F), GLY702 (2.5 Å — G702S!), SER821 (3.4 Å — long-range), GLY780 (3.6 Å — near V779/D801 region).

R703C sits in the dense 702-708 cluster (with G702S, F704, V707F, R708L, R708C). Loss of R703 charge + thiol introduction. ΔΔG essentially neutral; AM 0.471 below threshold; multi-phenotype does not resolve it (ClinVar: conflicting submissions).

Amino-acid chemistry
Arginine (R) → Cysteine (C) — charge loss + thiol introduction.
Position in the protein
C-terminal lumenal domain · position 703 (pLDDT 89).

Druggability Assessment

Category 4 — Stable Fold, Function Disrupted (AM under-call). ΔΔG ≈ 0. AlphaMissense 0.471 below threshold but three documented phenotypes confirm pathogenicity.

Mechanism: charge loss + thiol in the dense 702-708 cluster. Therapeutic: same multi-variant cluster.

Why this matters

R703C extends the 702-708 multi-variant cluster — six Atlas variants now converge here.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the R703C 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 R703C PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Topological domain653869 · Lumenal
Natural variant703703 · in DFNA6; dbSNP:rs1323852277