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9 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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MTSS2
MTSS I-BAR domain containing 2
Chromosome 16 Β· 16q22.1
NCBI Gene: 92154Ensembl: ENSG00000132613.16HGNC: HGNC:25094UniProt: Q765P7
21PubMed Papers
21Diseases
0Drugs
2Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
activation of GTPase activitycellular response to platelet-derived growth factor stimulusGTPase activator activityprotein bindingintellectual developmental disorder with ocular anomalies and distinctive facial featuressyndromic intellectual disabilityintelligencemicrocephaly
✦AI Summary

MTSS2 is a membrane-deforming cytoskeletal regulator encoding an I-BAR domain protein (also called ABBA/MTSS1L) highly expressed in the central nervous system with critical roles in neurodevelopment and cell dynamics. Functionally, MTSS2 promotes plasma membrane dynamics by activating the small GTPase Rac1, potentiating PDGF-mediated formation of membrane ruffles and lamellipodia in fibroblasts 1. The protein localizes to edges of membrane protrusions and facilitates dendritic spine formation, particularly in GABAergic inhibitory neurons such as parvalbumin-positive interneurons 2. During neurogenesis, MTSS2 localizes to the cleavage furrow where it recruits the scaffolding protein Nedd9 and positively regulates RhoA activity, ensuring proper mitotic progression in neuronal progenitor cells 3. MTSS2 dysfunction causes intellectual developmental disorder with ocular anomalies and distinctive facial features (OMIM#620086), predominantly caused by the recurrent de novo variant c.2011C>T (p.Arg671Trp) 4. This variant functions as a partial loss-of-function and dominant-negative allele, impairing Rac1 activation and synaptic transmission 4. Clinical manifestations include developmental delay, mild intellectual disability, microcephaly, facial dysmorphisms, seizures, hypotonia, learning disabilities, and multisystem involvement including cardiac and renal anomalies 56. MTSS2 represents a predominantly neurological but multisystem disorder affecting cognitive development and neural function.

Sources cited
1
MTSS2 localizes to cleavage furrow, recruits Nedd9, regulates RhoA activity during mitosis, and the R671W variant causes microcephaly and intellectual disability
PMID: 40698928
2
MTSS2 recurrent variant p.Arg671Trp causes intellectual developmental disorder with developmental delay, microcephaly, dysmorphisms, and multisystem comorbidities
PMID: 39890443
3
ABBA/MTSS2 is highly expressed in GABAergic inhibitory neurons, localizes to membrane protrusions, facilitates dendritic spine formation and filopodia initiation
PMID: 41359239
4
MTSS2-related neurodevelopmental disorder caused by recurrent p.Arg671Trp variant presents with intellectual disability, ocular anomalies, and expanded phenotype including autism and gastrointestinal anomalies
PMID: 37657631
5
Abba/MTSS2 promotes PDGF-mediated membrane ruffles and lamellipodia through Rac1 activation and GTPase activity enhancement
PMID: 20875796
6
MTSS2 c.2011C>T variant is a partial loss-of-function and dominant-negative allele causing syndromic intellectual disability with developmental delay and dysmorphisms
PMID: 36067766
7
ABBA/MTSS2 promotes cell spreading through Rac1 activation and is activated by tyrosine phosphorylation in response to cell spreading stimuli
PMID: 23060091
Disease Associationsβ“˜21
intellectual developmental disorder with ocular anomalies and distinctive facial featuresOpen Targets
0.46Moderate
syndromic intellectual disabilityOpen Targets
0.31Weak
intelligenceOpen Targets
0.31Weak
microcephalyOpen Targets
0.26Weak
Intellectual disabilityOpen Targets
0.26Weak
Global developmental delayOpen Targets
0.26Weak
HypotoniaOpen Targets
0.26Weak
MODYOpen Targets
0.07Suggestive
skull disorderOpen Targets
0.06Suggestive
Abnormality of the skeletal systemOpen Targets
0.05Suggestive
pancreatic carcinomaOpen Targets
0.05Suggestive
maturity-onset diabetes of the young type 3Open Targets
0.05Suggestive
maturity-onset diabetes of the young type 10Open Targets
0.04Suggestive
hypoparathyroidism, familial isolated, 2Open Targets
0.04Suggestive
diabetes mellitus, permanent neonatal 4Open Targets
0.04Suggestive
hyperproinsulinemiaOpen Targets
0.04Suggestive
diabetes mellitus, transient neonatal, 3Open Targets
0.04Suggestive
maturity-onset diabetes of the young type 13Open Targets
0.04Suggestive
permanent neonatal diabetes mellitus 1Open Targets
0.04Suggestive
Glycogen storage disease due to hepatic glycogen synthase deficiencyOpen Targets
0.04Suggestive
Intellectual developmental disorder with ocular anomalies and distinctive facial featuresUniProt
Pathogenic Variants2
NM_138383.3(MTSS2):c.2011C>T (p.Arg671Trp)Pathogenic
MTSS2-related neurodevelopmental disorder|Intellectual developmental disorder with ocular anomalies and distinctive facial features|Syndromic intellectual disability|not provided|not specified
β˜…β˜…β˜†β˜†2026β†’ Residue 671
NM_138383.3(MTSS2):c.1790C>T (p.Thr597Met)Likely pathogenic
Iron deposition in globus pallidus;Microcephaly;Intellectual disability;Hypotonia;Global developmental delay
β˜†β˜†β˜†β˜†2014β†’ Residue 597
View on ClinVar β†—
Related Genes
VAC14Protein interaction78%BAIAP2L2Shared pathway29%ARHGAP42Shared pathway25%RALGAPA2Shared pathway20%RALGAPBShared pathway20%SH3GLB2Shared pathway20%
Tissue Expression6 tissues
Brain
100%
Liver
48%
Heart
20%
Ovary
14%
Lung
14%
Bone Marrow
10%
Gene Interaction Network
Click a node to explore
MTSS2VAC14BAIAP2L2ARHGAP42RALGAPA2RALGAPBSH3GLB2
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q765P7
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.66LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.48 [0.35–0.66]
RankingsWhere MTSS2 stands among ~20K protein-coding genes
  • #13,961of 20,598
    Most Researched21
  • #4,549of 5,498
    Most Pathogenic Variants2
  • #4,856of 17,882
    Most Constrained (LOEUF)0.66
Genes detectedMTSS2
Sources retrieved9 papers
Response timeβ€”
πŸ“„ Sources
9β–Ό
1
Identification of novel human microcephaly-linked protein
PMID: 40698928
Elife Β· 2025
1.00
2
MTSS2 -Related Disorder: Refining the Phenotype in Four New Cases and Literature Review.
PMID: 39890443
Am J Med Genet A Β· 2025
0.89
3
Expression, Subcellular Localization, and Mechanistic Analysis of Intellectual Disability Syndrome Protein ABBA.
PMID: 41359239
Mol Neurobiol Β· 2025
0.78
4
MTSS2-related neurodevelopmental disorder: Further delineation of the phenotype.
PMID: 37657631
Eur J Med Genet Β· 2023
0.67
5
A large-scale genome-wide cross-trait analysis reveals shared genetic architecture between Alzheimer's disease and gastrointestinal tract disorders.
PMID: 35851147
Commun Biol Β· 2022
0.56