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10 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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MTMR1
myotubularin related protein 1
Chromosome X · Xq28
NCBI Gene: 8776Ensembl: ENSG00000063601.17HGNC: HGNC:7449UniProt: B7Z3D5
50PubMed Papers
20Diseases
0Drugs
0Pathogenic Variants
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
phosphatidylinositol-3-phosphate phosphatase activityprotein bindingprotein homodimerization activityphosphatidylinositol-3,5-bisphosphate 3-phosphatase activitypost-traumatic stress disorderBlackfan-Diamond anemiaThrombocytopeniainosine triphosphatase deficiency
✦AI Summary

MTMR1 is a lipid phosphatase belonging to the myotubularin family that catalyzes dephosphorylation of phosphatidylinositol 3-phosphate (PI(3)P) to phosphatidylinositol, and can also convert phosphatidylinositol 3,5-bisphosphate (PI(3,5)P2) to phosphatidylinositol 5-phosphate 12. Structurally, MTMR1 contains conserved pleckstrin homology (PH)-GRAM and protein tyrosine phosphatase (PTP) domains, with substrate specificity determined by strictly conserved residues in the C(X)5R motif 2. The gene arose through duplication from MTM1, the causative gene in X-linked myotubular myopathy, and is located 20 kb distal to MTM1 on Xq28 3. MTMR1 plays a role in muscle differentiation and myogenesis, with a muscle-specific isoform that is induced during myogenesis in vitro and in vivo 4. In myotonic dystrophy type 1 (DM1) and type 2 (DM2), MTMR1 alternative splicing is aberrantly regulated, correlating with impaired muscle fiber maturation and abnormal CUG-binding protein activity 54. While MTMR2 can partially compensate for myotubularin loss in X-linked myotubular myopathy therapy, MTMR1 cannot 6, suggesting functional specialization within the myotubularin family.

Sources cited
1
MTMR1 dephosphorylates PI(3)P to phosphatidylinositol
PMID: 11733541
2
MTMR1 crystal structure reveals PH-GRAM and PTP domains; substrate specificity for PI(3)P and PI(3,5)P2
PMID: 27018598
3
MTMR1 genomic organization, location 20 kb distal to MTM1 on Xq28, arose by gene duplication
PMID: 9828128
4
MTMR1 has muscle-specific isoforms induced during myogenesis; aberrant splicing in congenital myotonic dystrophy
PMID: 12217958
5
MTMR1 aberrant expression in DM1 and DM2 correlates with impaired muscle fiber maturation
PMID: 20685272
6
MTMR1 cannot functionally compensate for MTM1 loss in myotubular myopathy, unlike MTMR2
PMID: 29408998
7
MTMR1 splicing is corrected by CRISPR-Cas9 repeat excision in DM1 cardiomyocytes
PMID: 34371182
Disease Associationsⓘ20
post-traumatic stress disorderOpen Targets
0.09Suggestive
Blackfan-Diamond anemiaOpen Targets
0.08Suggestive
ThrombocytopeniaOpen Targets
0.07Suggestive
inosine triphosphatase deficiencyOpen Targets
0.07Suggestive
autosomal dominant macrothrombocytopeniaOpen Targets
0.06Suggestive
alpha thalassemia-intellectual disability syndrome type 1Open Targets
0.06Suggestive
Alpha-thalassemia - intellectual disability syndrome linked to chromosome 16Open Targets
0.06Suggestive
thrombocytopenia 4Open Targets
0.06Suggestive
macrothrombocytopenia, isolated, 2, autosomal dominantOpen Targets
0.06Suggestive
Beta-thalassemia - X-linked thrombocytopeniaOpen Targets
0.05Suggestive
beta-thalassemia-X-linked thrombocytopenia syndromeOpen Targets
0.05Suggestive
hemolytic anemia due to adenylate kinase deficiencyOpen Targets
0.05Suggestive
Heinz body anemiaOpen Targets
0.05Suggestive
autosomal dominant sideroblastic anemiaOpen Targets
0.05Suggestive
Constitutional sideroblastic anemiaOpen Targets
0.05Suggestive
delta-beta-thalassemiaOpen Targets
0.05Suggestive
Hereditary persistence of fetal hemoglobin - beta-thalassemiaOpen Targets
0.05Suggestive
hereditary persistence of fetal hemoglobin-sickle cell disease syndromeOpen Targets
0.05Suggestive
IRIDA syndromeOpen Targets
0.05Suggestive
Pyruvate kinase hyperactivityOpen Targets
0.05Suggestive
Pathogenic Variants
No pathogenic variants reported on ClinVar for this gene.
View on ClinVar ↗
Related Genes
FRG2Protein interaction99%PIKFYVEProtein interaction94%PIP4K2AProtein interaction94%PIP4K2BProtein interaction94%INPP4AProtein interaction94%PIK3C2AProtein interaction94%
Tissue Expression6 tissues
Brain
100%
Bone Marrow
94%
Lung
86%
Heart
81%
Ovary
78%
Liver
67%
Gene Interaction Network
Click a node to explore
MTMR1FRG2PIKFYVEPIP4K2APIP4K2BINPP4APIK3C2A
PROTEIN STRUCTURE
Preparing viewer…
PDB5C16 · 2.07 Å · X-ray
View on RCSB ↗
Constraintⓘ
LOEUFⓘ
0.49Moderately Constrained
pLIⓘ
0.99Intolerant
Observed/Expected LoF0.33 [0.23–0.49]
RankingsWhere MTMR1 stands among ~20K protein-coding genes
  • #8,847of 20,598
    Most Researched50
  • #2,923of 17,882
    Most Constrained (LOEUF)0.49 · top quartile
Genes detectedMTMR1
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
Genomic organization of a 225-kb region in Xq28 containing the gene for X-linked myotubular myopathy (MTM1) and a related gene (MTMR1).
PMID: 9828128
Genomics · 1998
1.00
2
Crystal Structure of Human Myotubularin-Related Protein 1 Provides Insight into the Structural Basis of Substrate Specificity.
PMID: 27018598
PLoS One · 2016
0.90
3
Analysis of MTMR1 expression and correlation with muscle pathological features in juvenile/adult onset myotonic dystrophy type 1 (DM1) and in myotonic dystrophy type 2 (DM2).
PMID: 20685272
Exp Mol Pathol · 2010
0.80
4
Muscle-specific alternative splicing of myotubularin-related 1 gene is impaired in DM1 muscle cells.
PMID: 12217958
Hum Mol Genet · 2002
0.70
5
MTM1 mutations in X-linked myotubular myopathy.
PMID: 10790201
Hum Mutat · 2000
0.60