SMCR8

Chr 17

SMCR8-C9orf72 complex subunit

Also known as: DENND8A

SMCR8 encodes a component of the C9orf72-SMCR8 complex that functions as a guanine nucleotide exchange factor and regulates autophagy by promoting RAB GTPase activation and inhibiting ULK1 kinase activity. Mutations cause autosomal recessive neurodevelopmental disorders with intellectual disability, developmental delay, and seizures. The gene shows moderate constraint against loss-of-function variants (LOEUF 0.654), suggesting some tolerance for such mutations.

OMIMResearchSummary from RefSeq, UniProt
LOEUF 0.65
Clinical SummarySMCR8
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
113 unique Pathogenic / Likely Pathogenic· 144 VUS of 272 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.65LOEUF
pLI 0.002
Z-score 2.84
OE 0.37 (0.230.65)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
-1.03Z-score
OE missense 1.13 (1.051.21)
574 obs / 508.9 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.37 (0.230.65)
00.351.4
Missense OE1.13 (1.051.21)
00.61.4
Synonymous OE1.28
01.21.6
LoF obs/exp: 9 / 24.0Missense obs/exp: 574 / 508.9Syn Z: -3.23

ClinVar Variant Classifications

272 submitted variants in ClinVar

Classification Summary

Pathogenic113
VUS144
Likely Benign5
Benign1
113
Pathogenic
144
VUS
5
Likely Benign
1
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
113
0
113
Likely Pathogenic
0
0
0
0
0
VUS
0
141
3
0
144
Likely Benign
0
2
1
2
5
Benign
0
1
0
0
1
Total01441172263

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

View in ClinVar →

Protein Context — Lollipop Plot

SMCR8 · 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 →
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC