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P428R

Category 3/4 — Most DruggablePathogenicTransmembrane · predictedEditorial
ProlineArginine at position 428 · TM4 (427-447), helical transmembrane · WFS1 (Wolframin)

Proline → Arginine at position 428 inside wolframin's fourth transmembrane helix (TM4). ClinVar Pathogenic. AlphaMissense 0.920, DynaMut2 ΔΔG -0.22 kcal/mol (destabilising). A proline-removal variant in a TM helix — opposite mechanism to L543P-type variants.

Interactive 3D Structure

Wild-type reference
Wild-type P428 — hydrogen bond to E431
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DynaMut2 mutant · P428R
Mutant R428 — hydrogen bond to E431 lost (5 contacts lost)
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Bond changes · DynaMut2 interaction analysis

5 lost0 gained5 preserved
Interaction typeWild-type partnerMutant partnerStatus
Hydrogen bondE431Lost
Hydrogen bondL432L432Preserved
Polar contactY351Lost
Polar contactS430Lost
Polar contactE431E431Preserved
Polar contactL432L432Preserved
Van der WaalsS430Lost
Van der WaalsL432L432Preserved
HydrophobicE431E431Preserved
HydrophobicL432Lost

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
-0.22kcal/mol
Destabilising — mild
AlphaMissense
0.920
LPath
AlphaFold pLDDT
89
model confidence
Schema
Cat 3/4
Category 3/4 — Most Druggable
ΔΔG confidence: Reduced confidence · Transmembrane
  • transmembrane position; predictor benchmarked on soluble proteins

This variant sits in the transmembrane band. The stability value was computed with a predictor benchmarked on water-soluble proteins. Published benchmarks of ddG predictors on membrane proteins report correlation below 0.4. Treat the magnitude as unreliable and the sign as weak evidence only.

Do not rank this value against a variant from a different region on ΔΔG alone.

Clinical Evidence

ClinVar classificationPathogenic
Review statuscriteria provided, single submitter
Associated conditions(no specific conditions catalogued for P428R — ClinVar Pathogenic by review evidence)
InheritanceInheritance not specified. ClinVar Pathogenic.
Population frequency (gnomAD v4)Ultra-rare · AF 0.00048%
cDNA changec.1283C>G
ClinVar accessionVCV002203517
Last evaluated2024/02/23 00:00

Observed at very low frequency in gnomAD.

Classified for1★ unverified submission

ClinVar classifies this variant as Pathogenic, but does not state what it is classified for. ClinVar records the condition only as "not provided". A classification without a phenotype and an inheritance mode cannot be read as a statement about Wolfram syndrome risk or severity.

  • 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★ single submitter. ClinVar "conditions" is the union of all submissions; 0–1★ entries are submitted conditions, not documented phenotypes.

Population frequency
Global (all ancestries)
AF 0.00048% · 7 / 1,461,816 alleles
Homozygotes
0
Highest-frequency population
European (non-Finnish) · AF 0.00063%

Highest in European (non-Finnish): AF 0.00063% (7 of 1,112,012 alleles), in line with the global figure.

No homozygotes reported in gnomAD v4. For a recessive allele this is expected and is not, on its own, evidence either way.

Ancestry groupAllele freq.AC / ANHom.Carrier est.
European (non-Finnish)0.00063%7 / 1,112,0120~1 in 79430

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 428 sits at the start of TM4. The AlphaFold model places P428 within 5 Å of ILE427 (2.5 Å), CYS429 (2.5 Å), SER430 (4.5 Å), and GLU431 (4.9 Å). The wild-type proline at 428 likely defines the helix-initiation geometry of TM4, providing a controlled bend or break at the start of the membrane-spanning segment.

Replacing proline with arginine has the same backbone-flexibility-gain as P504L (removed proline kink) but with a substantial added complication: a positive charge is introduced near the membrane-water interface. At position 428 — right at the start of TM4 — the arginine side chain can extend toward the lumenal interface where its charge is favorable, but the lost helix-initiation geometry remains a structural problem.

The |ΔΔG| of 0.22 indicates the fold absorbs the substitution. AlphaMissense's 0.920 score captures the functional consequence — TM4's insertion register shifts, the C429 immediately downstream (a possible disulfide-capable residue) is now in a different local geometry, and the E431 contact (4.9 Å) is perturbed.

Notably, the same E431 residue appears as a neighbor in the A559D atlas card — E431 sits between the connecting loop containing R558 and TM4 containing P428. The position is structurally connective in the lumenal-facing region.

Amino-acid chemistry
Proline (P) → Arginine (R) — rigid helix-breaking residue replaced by a large positively-charged residue. Loss of backbone constraint plus introduction of charge into the bilayer.
Position in the protein
TM4 (residues 427–447) · position 428 is at the very start of TM4, in the membrane-water interface region (pLDDT 89).

Druggability Assessment

Category 3/4 — Most Druggable. |ΔΔG| = 0.22 kcal/mol — fold survives. AlphaMissense 0.920 confirms severe functional consequence.

The mechanism combines loss of proline-defined helix-initiation geometry plus charge introduction at the membrane-water interface. Therapeutic strategy: site-directed small molecules at the TM4 N-terminal region.

The E431 contact suggests this position may be structurally connected to the R558-E431 microregion that A559D perturbs. Combined drug discovery in both regions has overlapping targets.

Why this matters

P428R is the proline-removal complement to the L→P variants elsewhere in the Atlas. The Atlas's mechanism vocabulary handles both proline introduction (helix break created) and proline removal (helix break eliminated) as distinct but related target categories.
Therapeutic Strategy Handoff · prediction

Feed this card to Wolfram Intelligence

Download the P428R 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 P428R PDF card ↓Strategies are AI-generated predictions, not validated therapeutics.

UniProt Domain Annotation

Chain1890 · Wolframin
Transmembrane427447 · Helical