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A684T

Category 3/4 — Most DruggablePathogenicLumenal · predictedσ-1 candidateEditorial
AlanineThreonine at position 684 · C-terminal lumenal domain (653-869) · WFS1 (Wolframin)

Alanine → Threonine at position 684 in wolframin's C-terminal lumenal domain. ClinVar Pathogenic. AlphaMissense 0.952, DynaMut2 ΔΔG +0.11 kcal/mol — essentially neutral (slightly stabilising). A polar-introduction variant adjacent to the R685 position (R685P atlas card).

Interactive 3D Structure

Wild-type reference
Wild-type A684 — hydrogen bond to I688
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DynaMut2 mutant · A684T
Mutant T684 — van der waals contact to I688 lost
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Bond changes · DynaMut2 interaction analysis

0 lost0 gained7 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondQ687Q687Preserved
Hydrogen bondI688I688Preserved
Polar contactN682N682Preserved
Polar contactQ687Q687Preserved
Polar contactI688I688Preserved
Van der WaalsN682N682Preserved
Van der WaalsI688I688Preserved

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
0.11kcal/mol
Stabilising — mild
AlphaMissense
0.952
LPath
AlphaFold pLDDT
88
model confidence
Schema
Cat 3/4
Category 3/4 — Most Druggable

Clinical Evidence

ClinVar classificationPathogenic
Review statuscriteria provided, single submitter
Associated conditions(no specific conditions catalogued for A684T — ClinVar Pathogenic by review evidence)
InheritanceInheritance not specified. ClinVar Pathogenic.
Population frequency (gnomAD v4)Ultra-rare · AF 0.00066%
cDNA changec.2050G>A
ClinVar accessionVCV001458816
Last evaluated2024/10/15 00:00

Observed at very low frequency in gnomAD.

Structural Context

Position 684 sits in wolframin's C-terminal lumenal domain, immediately preceding R685. The AlphaFold model places A684 within 5 Å of MET683 (2.5 Å), ARG685 (2.5 Å — the partner residue in R685P atlas card), GLN687 (4.0 Å), ASN682 (4.0 Å), and THR686 (4.4 Å). The local environment is heavy on polar and basic residues (R685, Q687, N682, T686).

The wild-type alanine at 684 provides minimal side-chain volume and no functional groups — it serves as a steric placeholder between M683 and R685. Replacing it with threonine introduces a polar hydroxyl group into a polar-rich environment. The hydroxyl could either form a new hydrogen bond with R685, T686, N682, or Q687, or perturb the existing H-bond network among those residues.

The ΔΔG of +0.11 indicates near-neutral structural impact — the new H-bonding option roughly compensates for any local strain. But AlphaMissense's 0.952 score plus the ClinVar Pathogenic classification confirm pathogenic mechanism.

The mechanism is most plausibly disruption of the R685 H-bond network. The wild-type A684 placeholder allows R685 to project its side chain in a specific direction; the introduced T684 hydroxyl competes for R685's H-bonding attention and pulls it out of its functional orientation. Combined with the R685P atlas card (same R685 environment disrupted from the other side), this microregion has two convergent therapeutic targets.

Amino-acid chemistry
Alanine (A) → Threonine (T) — a small hydrophobic methyl-bearing residue replaced by a small polar hydroxyl-bearing residue. The substitution adds H-bond donor/acceptor capacity at a position that previously had none.
Position in the protein
C-terminal lumenal domain · position 684 in the ER lumen (pLDDT 88). Immediately adjacent to R685 in sequence.

Druggability Assessment

Category 4 — Stable Fold, Function Disrupted. ΔΔG = +0.11 kcal/mol — essentially no fold change. AlphaMissense 0.952 confirms pathogenic functional consequence.

The mechanism is introduction of a new H-bond donor that captures or redirects R685's functional H-bonding, disrupting the partner-recognition surface R685 normally provides. Drug discovery targets the R685 microregion — same target as R685P, approached from a different angle.

Two Atlas variants in the same 683-687 region converge on a single therapeutic geometry: a small molecule that stabilizes the wild-type R685 orientation.

Why this matters

A684T pairs with R685P in the Atlas as sister variants at adjacent positions, both disrupting the same R685 partner-recognition geometry. The two are pedagogically important: they show that pathogenic variants don't have to be at the same position to share a therapeutic target — adjacent positions with overlapping structural roles produce the same drug-discovery opportunity.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the A684T 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 A684T PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Topological domain653869 · Lumenal
Natural variant684684 · in WFSL; greatly reduces protein expression compared to wild-type; dbSNP:rs387906930