PEDS1

Chr 20

plasmanylethanolamine desaturase 1

Also known as: CarF, KUA, TMEM189

The protein is a plasmanylethanolamine desaturase that catalyzes plasmalogen biosynthesis in the endoplasmic reticulum, producing specialized glycerophospholipids involved in antioxidative and signaling mechanisms. Mutations cause autosomal recessive neurodevelopmental disorders with intellectual disability and seizures. The gene shows moderate constraint to loss-of-function variants, suggesting some tolerance to complete gene loss.

Summary from RefSeq, UniProt
Research Assistant →
1
Active trials
5
Pubs (1 yr)
10
P/LP submissions
0%
P/LP missense
0.67
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryPEDS1
Population Constraint (gnomAD)
Low constraint (pLI 0.01) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
10 unique Pathogenic / Likely Pathogenic· 9 VUS of 21 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.67LOEUF
pLI 0.008
Z-score 2.65
OE 0.36 (0.200.67)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
2.43Z-score
OE missense 0.53 (0.450.62)
112 obs / 211.4 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.36 (0.200.67)
00.351.4
Missense OE0.53 (0.450.62)
00.61.4
Synonymous OE1.17
01.21.6
LoF obs/exp: 7 / 19.7Missense obs/exp: 112 / 211.4Syn Z: -1.18
DN
0.6936th %ile
GOF
0.6444th %ile
LOF
0.2580th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median
GOFprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

21 submitted variants in ClinVar

Classification Summary

Pathogenic7
Likely Pathogenic3
VUS9
Likely Benign1
7
Pathogenic
3
Likely Pathogenic
9
VUS
1
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
7
0
7
Likely Pathogenic
0
0
3
0
3
VUS
1
6
2
0
9
Likely Benign
0
1
0
0
1
Benign
0
0
0
0
0
Total1712020

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

PEDS1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Literature
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Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC