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GeneE
50 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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MLH1
mutL homolog 1
Chromosome 3 · 3p22.2
NCBI Gene: 4292Ensembl: ENSG00000076242.18HGNC: HGNC:7127UniProt: A0A087WX20
1,109PubMed Papers
25Diseases
0Drugs
1,934Pathogenic Variants
FUNCTIONAL ROLE
DNA RepairHub GeneTumor Suppressor
RESEARCH IMPACT
Highly StudiedVariant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
mismatch repairenzyme activator activityprotein bindingmeiotic mismatch repairLynch syndromemismatch repair cancer syndrome 1colorectal cancerMuir-Torre syndrome
✦AI Summary

MLH1 (mutL homolog 1) is a core component of the post-replicative DNA mismatch repair (MMR) system that heterodimerizes with PMS2 to form MutLα complex 1. This complex is recruited to mismatches detected by MutSα or MutSβ and activates PMS2 endonuclease activity to introduce single-strand breaks, enabling exonuclease EXO1 to degrade the error-containing strand 2. MLH1 also heterodimerizes with MLH3 to form MutLγ, involved in meiotic recombination. MLH1 protein stability is dynamically regulated through ubiquitin-proteasome degradation, with E3 ligase UBR4 promoting degradation while deubiquitylase USP5 stabilizes the protein; PMS2 binding protects MLH1 from degradation 3. Beyond repair function, MLH1 participates in DNA damage signaling and is associated with longevity 4. Clinically, MLH1 dysfunction is paramount in Lynch syndrome and colorectal cancer pathogenesis. Reduced MLH1 gene expression correlates with increased colorectal cancer stage and progression 5. MLH1 promoter methylation-mediated silencing causes microsatellite instability in endometrial and colorectal cancers 6. Functional characterization reveals that missense variants predominantly affect the ATP-binding pocket and C-terminal domain 1, with MLH1 disruption now targetable for improved prime editing genome therapy 7.

Sources cited
1
MLH1 variants affect ATP-binding pocket and C-terminal domain; functional assays characterize pathogenic variants
PMID: 17510385
2
MLH1 dimerization with PMS2 can be targeted therapeutically; dominant-negative MLH1 inhibits mismatch repair
PMID: 40769155
3
Pooled prime editing platform screens MLH1 variants for loss-of-function; demonstrates functional assessment in endogenous context
PMID: 40120586
4
Reduced MLH1 gene expression in colorectal cancer patients correlates with tumor invasion and lymph node invasion
PMID: 39400169
5
MLH1 is associated with longevity; loss of MLH1 increases cancer incidence and apoptosis resistance
PMID: 16474933
6
MLH1 promoter methylation silencing causes high microsatellite instability in endometrial and colorectal cancers
PMID: 23880961
7
UBR4 E3 ligase promotes MLH1 degradation while USP5 deubiquitylase stabilizes it; PMS2 binding protects MLH1 from degradation
PMID: 39032648
Disease Associationsⓘ25
Lynch syndromeOpen Targets
0.85Strong
mismatch repair cancer syndrome 1Open Targets
0.80Strong
colorectal cancerOpen Targets
0.79Strong
Muir-Torre syndromeOpen Targets
0.76Strong
colon carcinomaOpen Targets
0.75Strong
Constitutional mismatch repair deficiency syndromeOpen Targets
0.70Strong
colonic neoplasmOpen Targets
0.70Moderate
malignant colon neoplasmOpen Targets
0.69Moderate
hereditary nonpolyposis colon cancerOpen Targets
0.68Moderate
endometrial carcinomaOpen Targets
0.64Moderate
neoplasmOpen Targets
0.62Moderate
hereditary neoplastic syndromeOpen Targets
0.58Moderate
Inherited cancer-predisposing syndromeOpen Targets
0.58Moderate
Non-polyposis Turcot syndromeOpen Targets
0.58Moderate
hereditary nonpolyposis colorectal carcinomaOpen Targets
0.56Moderate
Hereditary breast and ovarian cancer syndromeOpen Targets
0.55Moderate
mismatch repair cancer syndromeOpen Targets
0.55Moderate
gastric cancerOpen Targets
0.55Moderate
ovarian carcinomaOpen Targets
0.54Moderate
Hereditary breast cancerOpen Targets
0.52Moderate
Colorectal cancerUniProt
Endometrial cancerUniProt
Lynch syndrome 2UniProt
Mismatch repair cancer syndrome 1UniProt
Muir-Torre syndromeUniProt
Pathogenic Variants1,934
NM_000249.4(MLH1):c.250A>G (p.Lys84Glu)Likely pathogenic
Lynch syndrome|Carcinoma of colon|Hereditary nonpolyposis colorectal neoplasms|not provided|Hereditary cancer-predisposing syndrome|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019→ Residue 84
NM_000249.3(MLH1):c.2104_2105delAGPathogenic
Lynch syndrome|Hereditary cancer-predisposing syndrome
★★★☆2019
NM_000249.4(MLH1):c.2104-2A>GLikely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019
NM_000249.4(MLH1):c.2153A>C (p.His718Pro)Likely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms|Muir-Torré syndrome|Hereditary cancer-predisposing syndrome
★★★☆2019→ Residue 718
NM_000249.4(MLH1):c.2103+1G>TLikely pathogenic
Lynch syndrome|Hereditary cancer-predisposing syndrome|not provided|Hereditary nonpolyposis colorectal neoplasms
★★★☆2019
NM_000249.4(MLH1):c.2103+1G>CLikely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms
★★★☆2019
NM_000249.4(MLH1):c.207+1_207+2delLikely pathogenic
Lynch syndrome|Gastric cancer
★★★☆2019
NM_000249.4(MLH1):c.207+1G>TLikely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms|Hereditary cancer-predisposing syndrome|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019
NM_000249.4(MLH1):c.2104-1G>TLikely pathogenic
Lynch syndrome
★★★☆2019
NM_000249.4(MLH1):c.1855G>C (p.Ala619Pro)Likely pathogenic
Lynch syndrome|Hereditary cancer-predisposing syndrome|Hereditary nonpolyposis colorectal neoplasms|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019→ Residue 619
NM_000249.4(MLH1):c.208-3C>GLikely pathogenic
Lynch syndrome|not provided|Hereditary nonpolyposis colorectal neoplasms|Hereditary cancer-predisposing syndrome|Colorectal cancer, hereditary nonpolyposis, type 2;Mismatch repair cancer syndrome 1;Muir-Torré syndrome|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019
NM_000249.4(MLH1):c.207+2T>CLikely pathogenic
Lynch syndrome|not provided|not specified|Hereditary cancer-predisposing syndrome|Hereditary nonpolyposis colorectal neoplasms|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019
NM_000249.4(MLH1):c.207+1G>ALikely pathogenic
Lynch syndrome|Hereditary cancer-predisposing syndrome|not provided|Hereditary nonpolyposis colorectal neoplasms|Colorectal cancer, hereditary nonpolyposis, type 2|Thyroid cancer, nonmedullary, 1
★★★☆2019
NM_000249.4(MLH1):c.2048T>C (p.Phe683Ser)Likely pathogenic
Lynch syndrome|not provided|Hereditary cancer-predisposing syndrome|Hereditary nonpolyposis colorectal neoplasms|Carcinoma of colon|Colorectal cancer, hereditary nonpolyposis, type 2|Hereditary nonpolyposis colon cancer
★★★☆2019→ Residue 683
NM_000249.4(MLH1):c.1668-1G>ALikely pathogenic
Lynch syndrome|Hereditary cancer-predisposing syndrome|Hereditary nonpolyposis colorectal neoplasms|not provided|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019
NM_000249.4(MLH1):c.143A>C (p.Gln48Pro)Likely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms|Colorectal cancer, hereditary nonpolyposis, type 2|Hereditary cancer-predisposing syndrome
★★★☆2019→ Residue 48
NM_000249.4(MLH1):c.1896+1G>ALikely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms|Hereditary cancer-predisposing syndrome|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019
NM_000249.4(MLH1):c.1664T>C (p.Leu555Pro)Likely pathogenic
Lynch syndrome|Hereditary cancer-predisposing syndrome|Colorectal cancer, hereditary nonpolyposis, type 2
★★★☆2019→ Residue 555
NM_000249.4(MLH1):c.1731+3A>TLikely pathogenic
Lynch syndrome
★★★☆2019
NM_000249.4(MLH1):c.1731+2T>GLikely pathogenic
Lynch syndrome|Hereditary nonpolyposis colorectal neoplasms|Colorectal cancer, hereditary nonpolyposis, type 2|Hereditary cancer-predisposing syndrome
★★★☆2019
View on ClinVar ↗
Related Genes
ERCC4Protein interaction100%XPAProtein interaction100%RFC4Protein interaction100%PCNAProtein interaction100%ERCC3Protein interaction99%XRCC6Protein interaction97%
Tissue Expression6 tissues
Heart
100%
Bone Marrow
58%
Lung
45%
Liver
40%
Brain
38%
Ovary
37%
Gene Interaction Network
Click a node to explore
MLH1ERCC4XPARFC4PCNAERCC3XRCC6
PROTEIN STRUCTURE
Preparing viewer…
PDB6WBA · 2.15 Å · X-ray
View on RCSB ↗
Constraintⓘ
LOEUFⓘ
0.64LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.48 [0.36–0.64]
RankingsWhere MLH1 stands among ~20K protein-coding genes
  • #136of 20,598
    Most Researched1,109 · top 1%
  • #15of 5,498
    Most Pathogenic Variants1,934 · top 1%
  • #4,558of 17,882
    Most Constrained (LOEUF)0.64
Genes detectedMLH1
Sources retrieved50 papers
Response time—
📄 Sources
50▼
1
Functional analysis of human MLH1 variants using yeast and in vitro mismatch repair assays.
PMID: 17510385
Cancer Res · 2007
1.00
2
AI-generated MLH1 small binder improves prime editing efficiency.
PMID: 40769155
Cell · 2025
0.90
3
Expression of Fhit, Mlh1, and P53 protein in human gallbladder carcinoma.
PMID: 12969785
Cancer Lett · 2003
0.86
4
PMID: 40458404
Front Immunol · 2025
0.84
5
It's prime time for multiplexed prime editing.
PMID: 40209678
Cell Genom · 2025
0.82