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L829P

Category 3/4 — Most DruggablePathogenic/Likely pathogenicLumenal · predictedσ-1 candidateEditorial
LeucineProline at position 829 · C-terminal lumenal domain (653-869) · WFS1 (Wolframin)

Leucine → Proline at position 829 in wolframin's C-terminal lumenal domain. ClinVar Pathogenic/Likely pathogenic with the full clinical spectrum — DFNA6 hearing loss, type 2 diabetes, inborn genetic diseases. AlphaMissense 0.997 (near-maximum), DynaMut2 ΔΔG -1.02 kcal/mol (destabilising). Another proline-introduction variant with substantial structural cost.

Interactive 3D Structure

Wild-type reference
Wild-type L829 — hydrogen bond to H696
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DynaMut2 mutant · L829P
Mutant P829 — hydrogen bond to G695 lost (12 contacts lost)
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Bond changes · DynaMut2 interaction analysis

12 lost5 gained5 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondL693Gained
Hydrogen bondE694Lost
Hydrogen bondG695Lost
Hydrogen bondH696Lost
Hydrogen bondL833Gained
Polar contactL693L693Preserved
Polar contactE694Lost
Polar contactG695Lost
Polar contactH696H696Preserved
Polar contactL833Gained
Polar contactF840Gained
CarbonylE694Lost
Van der WaalsE694Lost
Van der WaalsF840Gained
HydrophobicL664Lost
HydrophobicY669Lost
HydrophobicL693Lost
HydrophobicH696H696Preserved
HydrophobicV698Lost
HydrophobicW700Lost
HydrophobicL833L833Preserved
HydrophobicF840F840Preserved

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
-1.02kcal/mol
Destabilising — moderate
AlphaMissense
0.997
LPath
AlphaFold pLDDT
86
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 conditionsInborn genetic diseases; Autosomal dominant nonsyndromic hearing loss 6 (DFNA6); Type 2 diabetes mellitus
InheritanceAutosomal dominant pattern indicated by DFNA6 association.
Population frequency (gnomAD v4)Absent from gnomAD v4
cDNA changec.2486T>C
ClinVar accessionVCV000004521
Last evaluated2025/07/30 00:00

Not observed in ~730k individuals — consistent with a rare allele (ACMG PM2_supporting).

Structural Context

Position 829 sits in wolframin's C-terminal lumenal domain near the C-terminus. The AlphaFold model places L829 within 5 Å of GLU830 (2.4 Å), ILE828 (2.5 Å), HIS696 (3.9 Å — long-range across the fold), LEU833 (4.2 Å), and PHE840 (4.4 Å). The HIS696 contact at 3.9 Å is particularly notable: a residue 133 sequence positions away brought into structural contact through the folded geometry of the lumenal domain.

The wild-type leucine at 829 contributes branched hydrophobic packing into this multi-residue contact environment. Replacing it with proline introduces backbone constraint where flexibility was needed, breaks any α-helical character of the local region, and likely perturbs the long-range L829-H696 contact.

The |ΔΔG| of 1.02 is larger than the other proline-introduction variants in the Atlas (L723P 0.19, L402P +0.16, L543P 0.34) — reflecting that this position is more structurally constrained, and the proline kink has farther to propagate. AlphaMissense's 0.997 (near-maximum) confirms severe functional consequence.

The broad clinical spectrum (DFNA6 hearing loss, type 2 diabetes, inborn genetic diseases) reflects the multi-tissue impact of disrupting this fold-locking position.

Amino-acid chemistry
Leucine (L) → Proline (P) — flexible branched hydrophobic replaced by rigid helix-breaking residue. Same proline-introduction mechanism as L402P, L543P, L723P, L804P.
Position in the protein
C-terminal lumenal domain · position 829 near the C-terminus of the soluble domain (pLDDT 86).

Druggability Assessment

Category 3/4 — Most Druggable. |ΔΔG| = 1.02 kcal/mol — fold survives but with meaningful cost. AlphaMissense 0.997 + three documented phenotypes confirm severe functional consequence.

The mechanism is proline-induced backbone disruption at a position with long-range structural contact (HIS696 at 3.9 Å, 133 residues away in sequence). Therapeutic strategy: stabilize the L829-H696 long-range contact via small molecules engaging both sides of the contact.

The clinical breadth makes this a high-value docking target across multiple tissue contexts.

Why this matters

L829P's long-range H696 contact (133 sequence positions away) is exactly the kind of structural relationship invisible to sequence-based analysis. The Atlas surfaces it through the PDB neighbor extraction. Drug discovery aimed at the L829-H696 contact is a target that pre-atlas analysis could not have identified.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the L829P 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 L829P PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Topological domain653869 · Lumenal
Natural variant829829 · in DFNA6; dbSNP:rs104893883