RareResearch.AI
← Back to atlas

A716T

Category 3/4 — Most DruggablePathogenic/Likely pathogenicLumenal · predictedσ-1 candidateSource card
AlanineThreonine at position 716 · C-term lumenal (653-869) · WFS1 (Wolframin)

Interactive 3D Structure

Wild-type reference
Wild-type A716 — hydrogen bond to N714
Fullscreen ↗
DynaMut2 mutant · A716T
Mutant T716 — hydrogen bond contact to N714 lost
Fullscreen ↗

Bond changes · DynaMut2 interaction analysis

1 lost0 gained9 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondN714N714Preserved
Hydrogen bondA719A719Preserved
Hydrogen bondI720I720Preserved
Polar contactN714N714Preserved
Polar contactA719A719Preserved
Polar contactI720I720Preserved
Van der WaalsN714N714Preserved
Van der WaalsI720I720Preserved
HydrophobicI767Lost
HydrophobicF770F770Preserved

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
-0.80kcal/mol
Destabilising — mild
AlphaMissense
0.214
LBen
AlphaFold pLDDT
84
model confidence
Schema
Cat 3/4
Category 3/4 — Most Druggable

Clinical Evidence

ClinVar classificationPathogenic/Likely pathogenic
Review statuscriteria provided, multiple submitters, no conflicts
Associated conditionsRare genetic deafness; Monogenic hearing loss; not provided
Population frequency (gnomAD v4)Ultra-rare · AF 0.00048%
cDNA changec.2146G>A
ClinVar accessionVCV000004520
Last evaluated2026/01/24 00:00

Observed at very low frequency in gnomAD.

Full Variant Card

WFS1 Wolframin — A716T Variant Card

Molecular Atlas Pilot Variant · RareResearch.AI · Windsor Symposium Demo

Prepared: May 26, 2026 · Schema target: Category 4 (predicted)


Identity

FieldValue
VariantA716T
DNA changec.2146G>A
GeneWFS1
ProteinWolframin (890 aa)
UniProt IDO76024
ClinVar accessionVCV000004520
Amino acid changeA → T at position 716

Structural Context

FieldValue
AlphaFold modelAF-O76024-F1, v6
pLDDT at residue 71683.94
DomainC-terminal lumenal domain (653-869)
UniProt features at this position
  • Chain: 1-890 Wolframin
  • Topological domain: 653-869 Lumenal
  • Natural variant: 716-716 in DFNA6; dbSNP:rs28937893

Position 716 sits in the C-terminal lumenal domain, in a well-folded region (pLDDT 83.94). Alanine → Threonine is a small chemical change — adding a polar hydroxyl group where a hydrophobic methyl sat.


Computational Predictions

AlphaMissense

FieldValue
am_pathogenicity0.2145
am_classLBen
InterpretationLikely benign — note this disagrees with clinical signal

DynaMut2

FieldValue
Job ID177985952865
ΔΔG (kcal/mol)-0.8 kcal/mol (Destabilising)
Result URLhttps://biosig.lab.uq.edu.au/dynamut2/results_prediction/177985952865

Clinical Evidence

FieldValue
ClinVar classificationPathogenic/Likely pathogenic
Review statuscriteria provided, multiple submitters, no conflicts
Last evaluated2026/01/24 00:00
Associated conditionsRare genetic deafness; Monogenic hearing loss; not provided

Computational vs Clinical Tension

AlphaMissense scores A716T at 0.2145 (Likely Benign) — directly contradicting ClinVar's Pathogenic/Likely pathogenic classification supported by multiple independent submitters. This is the most important computational/clinical tension in the entire pilot set, and explains why the Atlas weights clinical evidence above predictions: AlphaMissense is trained primarily on loss-of-function and structure-destabilizing variants. A dominant-negative variant that disrupts oligomeric assembly without destabilizing the monomer is precisely the kind of mutation it can miss.


Phenotype focus

Autosomal dominant nonsyndromic low-frequency sensorineural hearing loss (DFNA6/14/38)

Carrier story

A716T is the textbook DFNA6 dominant hearing loss variant — a fundamentally different patient population than classical Wolfram. Onset is in childhood, progression is slow, and the phenotype is almost exclusively cochlear.

Mechanism hypothesis

Wolframin likely functions as a multimer in the ER membrane. A dominant variant like A716T poisons the multimer by inserting a malformed subunit, even when the monomer fold itself is fine. The schema's Category 4 (stable fold but functional site disrupted) is the right home for this mechanism.


Files in this folder

  • AF-O76024-F1-model_v6.pdb — AlphaFold structure
  • A716T_molstar_viewer.html — interactive 3D viewer (auto-loads and highlights position 716)
  • A716T_variant_card.md — this card
  • A716T_variant_card.html — demo-ready styled version

Final Schema Categorization

Category 4 — Most Druggable (functional disruption with stable fold)

DynaMut2 confirms the fold is intact (only -0.8 kcal/mol). Combined with A716T's documented dominant pattern of inheritance (DFNA6), this points to a dominant-negative mechanism — the monomer folds, but the multimer assembly is poisoned. AlphaMissense missed this because it's trained on monomer-destabilizing variants. The Atlas catches it via clinical evidence.


Every assumption documented. Every score sourced. The Atlas standard.

Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the A716T 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 A716T PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.