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E169K

Category 3/4 — Most DruggablePathogenic/Likely pathogenicCytoplasmic · predictedEditorial
GlutamateLysine at position 169 · N-terminal cytoplasmic domain (87-313) · WFS1 (Wolframin)

Glutamate → Lysine at position 169 in wolframin's N-terminal cytoplasmic domain. ClinVar Pathogenic/Likely pathogenic across both AD and AR WFS1-related disorders. AlphaMissense 0.948, DynaMut2 ΔΔG -0.39 kcal/mol (destabilising). A clean charge-flip variant with documented dual-inheritance impact.

Interactive 3D Structure

Wild-type reference
Wild-type E169 — ionic bond to R177
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DynaMut2 mutant · E169K
Mutant K169 — ionic bond to R177 lost (7 contacts lost)
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Bond changes · DynaMut2 interaction analysis

7 lost4 gained7 preserved
Interaction typeWild-type partnerMutant partnerStatus
Ionic bondR177Lost
Ionic bondK252Lost
Hydrogen bondQ165Q165Preserved
Hydrogen bondL166L166Preserved
Hydrogen bondE173E173Preserved
Hydrogen bondR174R174Preserved
Hydrogen bondK252Lost
Polar contactQ165Q165Preserved
Polar contactE173Gained
Polar contactR174R174Preserved
Polar contactR177Lost
Polar contactK252Lost
Van der WaalsS167Lost
Van der WaalsE173Gained
Van der WaalsR174Gained
Van der WaalsR177Lost
HydrophobicE173E173Preserved
HydrophobicK252Gained

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
-0.39kcal/mol
Destabilising — mild
AlphaMissense
0.948
LPath
AlphaFold pLDDT
87
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 conditionsAutosomal dominant and autosomal recessive WFS1-related disorders; Cataract 41
InheritanceBoth autosomal dominant and autosomal recessive WFS1-related disorders documented — a relatively rare profile that reflects the variant's mechanism affecting partner interactions in multiple cellular contexts.
Population frequency (gnomAD v4)Ultra-rare · AF 0.0050%
cDNA changec.505G>A
ClinVar accessionVCV000215376
Last evaluated2025/11/18 00:00

Observed at very low frequency in gnomAD.

Structural Context

Position 169 sits in wolframin's N-terminal cytoplasmic domain. The AlphaFold model places E169 within 5 Å of THR170 (2.4 Å), SER168 (2.5 Å), GLU173 (3.9 Å), LEU166 (4.2 Å), and GLN165 (4.2 Å). The local environment is polar-leaning, with a nearby second glutamate (E173) suggesting the wild-type E169 may contribute to a charged surface patch.

Replacing glutamate with lysine here reverses the charge sign at this position. Where the wild-type contributed a negative charge to the local electrostatic environment, the mutant contributes a positive one. The two glutamate residues E169 and E173 may have been forming a negatively-charged cytoplasmic surface patch — a recognition signature for a partner protein with a complementary positively-charged surface. Flipping E169 to lysine destroys that recognition surface and replaces it with one of opposite character.

The |ΔΔG| of 0.39 kcal/mol is modest because the fold itself accommodates the charge-flip — both glutamate and lysine are flexible polar residues. The structural cost is minor. But the functional cost — disrupted electrostatic recognition surface for partner proteins — is severe, captured by AlphaMissense's 0.948 score and the documented dual-inheritance clinical impact.

Amino-acid chemistry
Glutamate (E) → Lysine (K) — a negatively-charged carboxylate-bearing residue replaced by a positively-charged primary amine-bearing residue. The charge sign reverses completely; volume is roughly comparable.
Position in the protein
N-terminal cytoplasmic domain · position 169 sits in the cytosol-facing region with good AlphaFold confidence (pLDDT 86). The cytosolic environment accommodates charged side chains but the specific charge sign matters for partner interactions.

Druggability Assessment

Category 3/4 — Most Druggable. |ΔΔG| = 0.39 kcal/mol — fold survives. AlphaMissense 0.948 + dual-inheritance clinical impact confirm severe functional consequence.

The mechanism is charge-sign reversal at a cytoplasmic recognition surface. The lost negative charge plus the new positive charge together disrupt whatever partner-protein recognition the wild-type E169/E173 patch enabled.

Therapeutic strategy: this is a functional-site disruption rather than a fold problem. Site-directed small-molecule design at the recognition surface — restoring or compensating for the lost negatively-charged patch — is the rational vector. Alternatively, if the disrupted recognition target can be identified, indirect rescue via the partner protein.

Why this matters

E169K is one of the few Atlas variants with the unusual dual-inheritance ClinVar classification, suggesting the disrupted partner interaction operates in multiple cellular contexts (with different consequences in homozygous, compound heterozygous, and heterozygous settings). The charge-flip mechanism class — where the substitution reverses charge sign without changing volume much — is structurally invisible to ΔΔG analysis but clear to AlphaMissense and clinical evidence.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the E169K 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 E169K PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Region1321 · Interaction with ATP6V1A
Natural variant169169 · in WFS1; dbSNP:rs148953711