RareResearch.AI
← Back to atlas

A243V

Category 4 — Stable Fold, Function DisruptedConflictingCytoplasmic · predictedEditorial
AlanineValine at position 243 · N-terminal cytoplasmic domain (87-313) · WFS1 (Wolframin)

Alanine → Valine at position 243 in N-terminal cytoplasmic domain. ClinVar Conflicting including monogenic diabetes. AlphaMissense 0.27 (below threshold) — AM under-call. DynaMut2 ΔΔG -0.93.

Interactive 3D Structure

Wild-type reference
Wild-type A243 — hydrogen bond to D246
Fullscreen ↗
DynaMut2 mutant · A243V
Mutant V243 — hydrogen bond to D246 lost (2 contacts lost)
Fullscreen ↗

Bond changes · DynaMut2 interaction analysis

2 lost2 gained5 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondD246D246Preserved
Hydrogen bondF247F247Preserved
Polar contactD245Lost
Polar contactD246D246Preserved
Polar contactF247F247Preserved
Van der WaalsD245Lost
Van der WaalsF247Gained
HydrophobicQ194Q194Preserved
HydrophobicD246Gained

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
-0.93kcal/mol
Destabilising — mild
AlphaMissense
0.269
LBen
AlphaFold pLDDT
86
model confidence
Schema
Cat 4
Category 4 — Stable Fold, Function Disrupted
ΔΔG confidence: Standard confidence · Cytoplasmic

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 conditionsMonogenic diabetes; Inborn genetic diseases
InheritanceMonogenic diabetes.
Population frequency (gnomAD v4)Low frequency · AF 0.040%
cDNA changec.728C>T
ClinVar accessionVCV000215381
Last evaluated2025/11/16 00:00

Observed in the general population.

Classified for1★ unverified submission

ClinVar classifies this variant as Conflicting classifications of pathogenicity for WFS1-related diabetes (autosomal dominant); Ataxia / spastic ataxia (inheritance not specified); WFS1-related spectrum (unresolved mode) (dominant or recessive); Wolfram syndrome (classic) (autosomal recessive, OMIM 222300); Cataract 41 (autosomal dominant, OMIM 116400); Nonsyndromic hearing loss (DFNA6/14/38) (autosomal dominant, OMIM 600965); Wolfram-like syndrome (autosomal dominant, OMIM 614296). 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 diabetesautosomal dominantsubmitted as: Monogenic diabetes; Type 2 diabetes mellitus
  • Ataxia / spastic ataxiainheritance not specifiedsubmitted as: Hereditary ataxia
  • WFS1-related spectrum (unresolved mode)dominant or recessivesubmitted as: WFS1-Related Spectrum Disorders
  • Wolfram syndrome (classic)autosomal recessive · OMIM 222300submitted as: Wolfram syndrome 1
  • Cataract 41autosomal dominant · OMIM 116400submitted as: Cataract 41
  • Nonsyndromic hearing loss (DFNA6/14/38)autosomal dominant · OMIM 600965submitted as: Autosomal dominant nonsyndromic hearing loss 6; Autosomal dominant nonsyndromic hearing loss 6
  • 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 0.040% · 648 / 1,613,594 alleles
Homozygotes
1
Highest-frequency population
South Asian · AF 0.108%

Highest in South Asian: AF 0.108% (98 of 91,090 alleles), 2.7x the global figure. The global AF describes the general population, not the at-risk group.

1 homozygote reported in gnomAD v4 (1 South Asian). 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.
South Asian0.108%98 / 91,0901~1 in 460
Remaining individuals0.056%35 / 62,4980~1 in 890
European (non-Finnish)0.043%503 / 1,180,0200~1 in 1170
Admixed American0.0067%4 / 60,0260~1 in 7500
African / African American0.0067%5 / 75,0540~1 in 7510
Finnish0.0047%3 / 63,4560~1 in 10580

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 243 in cytoplasmic domain. Neighbors: LEU244 (2.5 Å), ILE242 (2.5 Å), LYS193 (4.0 Å — long-range contact!). The K193 long-range contact suggests A243 sits in a structural element bringing distant sequence positions into contact.

|ΔΔG| 0.93 substantial for conservative substitution. AM 0.27 under-call; monogenic diabetes does not resolve it (ClinVar: conflicting submissions).

Amino-acid chemistry
Alanine (A) → Valine (V) — small replaced by branched aliphatic. Conservative volume increase.
Position in the protein
N-terminal cytoplasmic domain · position 243 (pLDDT 86).

Druggability Assessment

Category 3/4 — Most Druggable (AM under-call). |ΔΔG| 0.93. AlphaMissense 0.27 below threshold and monogenic diabetes does not resolve it (ClinVar: conflicting submissions).

Mechanism: volume mismatch perturbing K193 long-range contact. Therapeutic: site-directed at the 193-243 cross-fold geometry.

Why this matters

A243V demonstrates a long-range 50-residue contact (K193). The Atlas's neighbor analysis surfaces these cross-domain interactions.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the A243V 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 A243V PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Region1321 · Interaction with ATP6V1A