The RNASEK protein is an endoribonuclease that regulates vacuolar H+-ATPase activity, which is essential for acidifying intracellular compartments and receptor-mediated endocytosis. Mutations cause MEND syndrome (mental retardation, enteropathy, deafness, peripheral neuropathy, ichthyosis, and keratoderma), a severe multisystem disorder with autosomal recessive inheritance. The condition involves intellectual disability, gastrointestinal dysfunction, hearing loss, and skin abnormalities with early childhood onset.

OMIMResearchSummary from RefSeq, UniProt
MultiplemechanismLOEUF 0.78
Clinical SummaryRNASEK
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.50) — some intolerance to loss-of-function variants.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.78LOEUF
pLI 0.504
Z-score 1.91
OE 0.16 (0.060.78)
Moderately constrained

Typical tolerance to LoF variation

Missense Constraint
0.50Z-score
OE missense 0.85 (0.701.03)
71 obs / 83.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.16 (0.060.78)
00.351.4
Missense OE0.85 (0.701.03)
00.61.4
Synonymous OE1.01
01.21.6
LoF obs/exp: 1 / 6.1Missense obs/exp: 71 / 83.8Syn Z: -0.03
DN
0.6937th %ile
GOF
0.73top 25%
LOF
0.3259th %ile

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

GOFprediction above median
DNprediction 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

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

RNASEK · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
Open Research Assistant →