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10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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MAB21L1
mab-21 like 1
Chromosome 13 Β· 13q13.3
NCBI Gene: 4081Ensembl: ENSG00000180660.10HGNC: HGNC:6757UniProt: B2R805
24PubMed Papers
21Diseases
0Drugs
8Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindingeye developmentanatomical structure morphogenesisnucleuscerebellar, ocular, craniofacial, and genital syndromeIntellectual disabilityGlobal developmental delayAbnormality of the genital system
✦AI Summary

MAB21L1 is a putative nucleotidyltransferase required for embryonic development, particularly eye, cerebellar, craniofacial, and genital development 12. The protein contains a conserved nucleotidyltransferase fold structurally similar to cGAS 3, though in vitro nucleotidyltransferase activity has not been demonstrated 4. MAB21L1 binds single-stranded RNA 3 and associates with transcription factors regulating PAX6 (MEIS1, MEIS2, PBX1) and poly(A) RNA-binding proteins 4. During lens placode development, MAB21L1 modulates lens-specific gene expression (including Pitx3, Maf, and Sfrp2) and regulates DNA/nucleotide metabolic processes 5. Biallelic loss-of-function MAB21L1 mutations cause autosomal recessive cerebellar, ocular, craniofacial, and genital (COFG) syndrome with severe microphthalmia 5. Conversely, heterozygous missense variants at the Arg51 codon cause autosomal dominant microphthalmia and aniridia phenotypes via a gain-of-function mechanism 467. These Arg51 mutations reduce association with TBL1XR1 (NCoR complex component) and alter nuclear localization 47. MAB21L1 also regulates calvarial osteogenesis and bone resorption 8. Clinically, MAB21L1 variants account for approximately 1-3% of congenital microphthalmia cases and should be included in genetic testing panels for congenital eye disorders 7.

Sources cited
1
The protein contains a conserved nucleotidyltransferase fold structurally similar to cGAS , though in vitro nucleotidyltransferase activity has not been demonstrated .
PMID: 27271801
2
The protein contains a conserved nucleotidyltransferase fold structurally similar to cGAS , though in vitro nucleotidyltransferase activity has not been demonstrated .
PMID: 36413568
3
During lens placode development, MAB21L1 modulates lens-specific gene expression (including Pitx3, Maf, and Sfrp2) and regulates DNA/nucleotide metabolic processes .
PMID: 34779479
4
MAB21L1 also regulates calvarial osteogenesis and bone resorption .
PMID: 29156428
5
Conversely, heterozygous missense variants at the Arg51 codon cause autosomal dominant microphthalmia and aniridia phenotypes via a gain-of-function mechanism , , .
PMID: 36446583
Disease Associationsβ“˜21
cerebellar, ocular, craniofacial, and genital syndromeOpen Targets
0.71Strong
Intellectual disabilityOpen Targets
0.37Weak
Global developmental delayOpen Targets
0.37Weak
Abnormality of the eyeOpen Targets
0.37Weak
Abnormality of the genital systemOpen Targets
0.37Weak
Cerebellar hypoplasiaOpen Targets
0.37Weak
esophageal diseaseOpen Targets
0.30Weak
alcohol drinkingOpen Targets
0.26Weak
osteoarthritis, kneeOpen Targets
0.23Weak
androgenetic alopeciaOpen Targets
0.22Weak
medical procedureOpen Targets
0.20Weak
gastroesophageal reflux diseaseOpen Targets
0.19Weak
secondary malignant neoplasmOpen Targets
0.19Weak
genetic disorderOpen Targets
0.19Weak
hemiplegiaOpen Targets
0.19Weak
kidney transplantOpen Targets
0.17Weak
mathematical abilityOpen Targets
0.16Weak
Dupuytren ContractureOpen Targets
0.15Weak
neurodevelopmental disorder with or without early-onset generalized epilepsyOpen Targets
0.12Weak
smoking initiationOpen Targets
0.11Weak
Cerebellar, ocular, craniofacial, and genital syndromeUniProt
Pathogenic Variants8
NM_005584.5(MAB21L1):c.371C>T (p.Ser124Leu)Likely pathogenic
Cerebellar, ocular, craniofacial, and genital syndrome
β˜…β˜†β˜†β˜†2026β†’ Residue 124
NM_005584.5(MAB21L1):c.152G>A (p.Arg51Gln)Pathogenic
not provided
β˜…β˜†β˜†β˜†2022β†’ Residue 51
NM_005584.5(MAB21L1):c.737dup (p.Cys246fs)Likely pathogenic
Hypoplasia of scrotum
β˜…β˜†β˜†β˜†2014β†’ Residue 246
NM_005584.5(MAB21L1):c.840C>G (p.Tyr280Ter)Pathogenic
Cerebellar, ocular, craniofacial, and genital syndrome
β˜†β˜†β˜†β˜†2019β†’ Residue 280
NM_005584.5(MAB21L1):c.859del (p.Arg287fs)Pathogenic
Cerebellar, ocular, craniofacial, and genital syndrome
β˜†β˜†β˜†β˜†2019β†’ Residue 287
NM_005584.5(MAB21L1):c.841del (p.Glu281fs)Pathogenic
Cerebellar, ocular, craniofacial, and genital syndrome
β˜†β˜†β˜†β˜†2019β†’ Residue 281
NM_005584.5(MAB21L1):c.698A>C (p.Gln233Pro)Pathogenic
Cerebellar, ocular, craniofacial, and genital syndrome
β˜†β˜†β˜†β˜†2019β†’ Residue 233
NM_005584.5(MAB21L1):c.735dup (p.Cys246fs)Pathogenic
Cerebellar, ocular, craniofacial, and genital syndrome
β˜†β˜†β˜†β˜†2019β†’ Residue 246
View on ClinVar β†—
Related Genes
PBX1Protein interaction100%MEIS2Protein interaction99%LMX1BProtein interaction87%MEIS1Protein interaction87%NBEAProtein interaction82%FAM222AProtein interaction78%
Tissue Expression6 tissues
Heart
100%
Ovary
81%
Brain
32%
Lung
13%
Liver
8%
Bone Marrow
0%
Gene Interaction Network
Click a node to explore
MAB21L1PBX1MEIS2LMX1BMEIS1NBEAFAM222A
PROTEIN STRUCTURE
Preparing viewer…
PDB5EOM Β· 2.55 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.47Moderately Constrained
pLIβ“˜
0.99Intolerant
Observed/Expected LoF0.26 [0.15–0.47]
RankingsWhere MAB21L1 stands among ~20K protein-coding genes
  • #13,234of 20,598
    Most Researched24
  • #3,129of 5,498
    Most Pathogenic Variants8
  • #2,692of 17,882
    Most Constrained (LOEUF)0.47 Β· top quartile
Genes detectedMAB21L1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
MAB21L1 modulates gene expression and DNA metabolic processes in the lens placode.
PMID: 34779479
Dis Model Mech Β· 2021
1.00
2
Synchronized long-read genome, methylome, epigenome and transcriptome profiling resolve a Mendelian condition.
PMID: 39880924
Nat Genet Β· 2025
0.90
3
Missense Mutations in MAB21L1: Causation of Novel Autosomal Dominant Ocular BAMD Syndrome.
PMID: 36892533
Invest Ophthalmol Vis Sci Β· 2023
0.80
4
Monoallelic variants resulting in substitutions of MAB21L1 Arg51 Cause Aniridia and microphthalmia.
PMID: 36413568
PLoS One Β· 2022
0.70
5
Monoallelic missense variants in
PMID: 39016008
Ophthalmic Genet Β· 2024
0.60