HTRA1

Chr 10ADAR

HtrA serine peptidase 1

Also known as: ARMD7, CADASIL2, CARASIL, CARASIL2, HtrA, L56, ORF480, PRSS11

This serine protease regulates extracellular matrix proteins, insulin-like growth factor availability, and TGF-beta signaling pathways. Mutations cause cerebral arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL-like syndrome), which can follow either autosomal dominant or autosomal recessive inheritance patterns. The gene shows low constraint against loss-of-function variants, consistent with both recessive disease and the observation that some individuals may tolerate heterozygous mutations.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismAD/ARLOEUF 0.752 OMIM phenotypes
Clinical SummaryHTRA1
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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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
0.75LOEUF
pLI 0.000
Z-score 2.41
OE 0.43 (0.260.75)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
1.05Z-score
OE missense 0.81 (0.720.91)
195 obs / 241.1 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.43 (0.260.75)
00.351.4
Missense OE0.81 (0.720.91)
00.61.4
Synonymous OE1.06
01.21.6
LoF obs/exp: 9 / 20.9Missense obs/exp: 195 / 241.1Syn Z: -0.51
DN
0.7326th %ile
GOF
0.6443th %ile
LOF
0.3452th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative, gain-of-function and loss-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
LOF1 literature citation

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.

Literature Evidence

LOFAltogether, our findings strongly suggest that heterozygous HTRA1 stop codons are pathogenic through a haploinsufficiency mechanism.PMID:34270682

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

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

HTRA1 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
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