MLH1

Chr 3ARAD

mutL homolog 1

Also known as: COCA2, FCC2, HNPCC, HNPCC2, LYNCH2, MLH-1, MMRCS1, hMLH1

The MLH1 protein heterodimerizes with PMS2 to form MutL alpha, a key component of the DNA mismatch repair system that introduces single-strand breaks near DNA mismatches to initiate repair by exonucleases. Mutations cause Lynch syndrome, mismatch repair cancer syndrome, and Muir-Torre syndrome with both autosomal dominant and autosomal recessive inheritance patterns. The gene is highly constrained against loss-of-function variants (pLI near 0, LOEUF 0.575), reflecting its essential role in maintaining genomic stability.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismAR/ADLOEUF 0.573 OMIM phenotypes
Clinical SummaryMLH1
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Gene-Disease Validity (ClinGen)
Lynch syndrome · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

3 total gene-disease associations curated

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
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GeneReview available — MLH1
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.57LOEUF
pLI 0.000
Z-score 3.68
OE 0.37 (0.250.57)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
-0.30Z-score
OE missense 1.04 (0.961.13)
413 obs / 396.0 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.37 (0.250.57)
00.351.4
Missense OE1.04 (0.961.13)
00.61.4
Synonymous OE1.09
01.21.6
LoF obs/exp: 15 / 40.2Missense obs/exp: 413 / 396.0Syn Z: -0.83
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveMLH1-related Lynch syndromeLOFAD
definitiveMLH1-related Muir-Torre syndromeLOFAD
definitiveMLH1-related constitutional mismatch repair deficiency syndrome (CMMRD)LOFAR
DN
0.6938th %ile
GOF
0.5170th %ile
LOF
0.3549th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative, gain-of-function and loss-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median · 1 literature citation
GOF1 literature citation
LOF1 literature citation

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

DNAlternative splicing of hMLH1 in childhood acute lymphoblastic leukaemia and characterisation of the variably expressed Delta9/10 isoform as a dominant negative species.PMID:19767099
GOFWe also detected a gain-of-function mutation in the GRB7, MAP3K1, and MLH1 genes.PMID:34680380
LOFGenomic deletions in MSH2 or MLH1 are a frequent cause of hereditary non-polyposis colorectal cancer: identification of novel and recurrent deletions by MLPAPMID:14635101

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

MLH1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

MMR MutationSmall Bowel AdenomaSmall-bowel Adenocarcinoma

Small Bowel Capsule Endoscopy in Lynch Syndrome

RECRUITING
NCT07472686Assistance Publique - Hôpitaux de ParisStarted 2025-06-16
Basal Cell Carcinoma of Skin, Site UnspecifiedEpidermoid CarcinomaLynch Syndrome

Determining the Prevalence of Muir-Torre Syndrome in Patients With Lynch Syndrome

NOT YET RECRUITING
NCT07201012Phase NACentre Hospitalier Universitaire de NīmesStarted 2025-12
Sampling of suspected skin lesions (in accordance with good care practices).Constitution of a biobank
Pancreatic CancerPancreatic Ductal AdenocarcinomaPDAC

A Prospective Registry for Patients at High-Risk for Pancreatic Cancer

RECRUITING
NCT06151223Mayo ClinicStarted 2021-07-13
Bio-specimen Collection: BloodBio-specimen Collection: Pancreatic JuiceMRI
Solid TumorAdvanced Solid TumorMetastatic Cancer

KPMNG Study of MOlecular Profiling Guided Therapy Based on Genomic Alterations in Advanced Solid Tumors II

RECRUITING
NCT05525858Seoul National University Bundang HospitalStarted 2022-09-28
AlectinibAtezolizumabErlotinib
Polycythemia VeraEssential ThrombocythaemiaMyelofibrosis

Prevalence Of Germline Gene Mutations In Patients With Myeloproliferative Neoplasms With Family History

NOT YET RECRUITING
NCT06923670Phase NAFondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2025-05-21
NGS testingNGS analysis for mutations in genes involved in familial predisposition to hematological malignancies
Uveal Melanoma

Identification of New Candidate Genes for Hereditary Predisposition to Uveal Melanoma

RECRUITING
NCT06550674Phase NACentre Jean PerrinStarted 2024-10-29
Constitutional exome analysis
Lynch SyndromeEndometrial Cancer

EC_ItaLynch: Mainstreaming the Diagnosis of Lynch Syndrome

NOT YET RECRUITING
NCT06501417Fondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2024-08-01
mainstreaming
BRCA1 MutationPOLD1 Gene MutationCDKN2A Mutation

An Intervention to Increase Genetic Testing in Families Who May Share a Gene Mutation Related to Cancer Risk and An Intervention to Help Patients and Their Primary Care Providers Stay Up-to-date About Uncertain Genetic Test Results

RECRUITING
NCT05420064Phase NAMemorial Sloan Kettering Cancer CenterStarted 2022-12-01
Intervention Arm At-risk Relative/ARR ContactsMyGene PortalStandard of Care
Lynch SyndromeLynch Syndrome ILynch Syndrome II

Videocapsule Endoscopy in Lynch Syndrome

RECRUITING
NCT05704010Phase NASan Raffaele UniversityStarted 2018-11-01
Video capsule endoscopy
Breast Cancer RiskOvarian Cancer RiskCancer Gene Mutation

Population Based Germline Testing for Early Detection and Prevention of Cancer

RECRUITING
NCT07498829Phase NAQueen Mary University of LondonStarted 2025-12-18
Genetic testing for Cancer Susceptibility Genes (CSGs) (BRCA1, BRCA2, RAD51C, RAD51D, BRIP1, PALB2, MLH1, MSH2, MSH6) and personalised breast and ovarian cancer risk
Deleterious BARD1 Gene MutationDeleterious BRCA1 Gene MutationDeleterious BRCA2 Gene Mutation

Surgery in Preventing Ovarian Cancer in Patients With Genetic Mutations

ACTIVE NOT RECRUITING
NCT02760849Phase NAM.D. Anderson Cancer CenterStarted 2016-05-02
Laboratory Biomarker AnalysisOophorectomyQuality-of-Life Assessment
Hereditary Breast/Ovarian Cancer (brca1, brca2)Lynch SyndromeGenetic Variation

Patient Centered Clinical Decision Support for Hereditary Cancer Syndromes

ENROLLING BY INVITATION
NCT06914726Phase NAHealthPartners InstituteStarted 2025-07-09
Patient Centered Clinical Decision Support (PC-CDS)
Clinical Literature
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Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗