MAF1

Chr 8

MAF1 negative regulator of RNA polymerase III

MAF1 encodes a protein that represses RNA polymerase III-mediated transcription of tRNAs, 5S rRNA, and other small non-coding RNAs in response to nutritional and cellular stress conditions. Mutations cause autosomal dominant neurodevelopmental disorders with intellectual disability and multiple congenital anomalies. The gene is highly constrained against loss-of-function variants (pLI 0.95, LOEUF 0.35), indicating that complete loss of function is likely incompatible with normal development.

Summary from RefSeq, UniProt
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1
Active trials
12
Pubs (1 yr)
62
P/LP submissions
0%
P/LP missense
0.34
LOEUF· LoF intol.
LOF
Mechanism· predicted
Clinical SummaryMAF1
Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.95). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
61 unique Pathogenic / Likely Pathogenic· 33 VUS of 114 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available
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GeneReview available — MAF1
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.34LOEUF
pLI 0.948
Z-score 3.18
OE 0.07 (0.030.34)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
2.22Z-score
OE missense 0.48 (0.390.59)
69 obs / 143.9 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.07 (0.030.34)
00.351.4
Missense OE0.48 (0.390.59)
00.61.4
Synonymous OE1.55
01.21.6
LoF obs/exp: 1 / 13.7Missense obs/exp: 69 / 143.9Syn Z: -3.25
DN
0.3991th %ile
GOF
0.3292th %ile
LOF
0.64top 25%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · LOEUF 0.34

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

114 submitted variants in ClinVar

Classification Summary

Pathogenic56
Likely Pathogenic5
VUS33
Likely Benign4
Benign1
56
Pathogenic
5
Likely Pathogenic
33
VUS
4
Likely Benign
1
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
56
0
56
Likely Pathogenic
0
0
5
0
5
VUS
0
22
11
0
33
Likely Benign
0
3
0
1
4
Benign
0
0
0
1
1
Total02572299

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

View in ClinVar →

Protein Context — Lollipop Plot

MAF1 · 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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