MUTYH-Associated Polyposis

Mendelian MONDO:0012041 Pathograph 7 Show in embeddings browser hereditary cancer-predisposing syndrome

MUTYH-associated polyposis (MAP) is an autosomal recessive colorectal adenomatous polyposis caused by biallelic germline variants in MUTYH, the human homolog of bacterial mutY, which encodes the adenine DNA glycosylase of the base-excision-repair pathway. It is the exception that clarifies the rule among the polyposis syndromes, and its mechanism is deliberately NOT the Knudson two-hit route. MAP carriers inherit two damaged alleles of a repair gene rather than one damaged allele of a tumor suppressor; the resulting defect is a constitutional mutator phenotype rather than a constitutional loss of growth restraint. MUTYH excises adenine that DNA polymerase has misincorporated opposite 8-oxoguanine, the commonest oxidative base lesion. Without it, those mispairs are fixed as G:C to T:A transversions, and the tumor suppressor that actually initiates the adenoma — APC — is then inactivated somatically by exactly that mutational signature. The evidence for this chain is unusually direct: MUTYH was identified by noticing that a family with multiple colorectal adenomas but no germline APC mutation had tumors whose somatic APC mutations were overwhelmingly G:C to T:A transversions, and in confirmed biallelic carriers every somatic APC mutation found was of that type. Clinically MAP presents as multiple (typically 15 to a few hundred) colorectal adenomas, overlapping both attenuated and classic familial adenomatous polyposis, and its recognition matters because the recessive inheritance changes who in the family is at risk: siblings, not offspring, are the high-risk relatives.

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1
Inheritance
4
Pathophys.
4
Phenotypes
7
Pathograph
1
Genes
2
Medical Actions
1
Trials
1
References
👪

Inheritance

1
Autosomal recessive HP:0000007
MAP is inherited as an autosomal recessive trait; both MUTYH alleles must be damaged. This is the single most important practical difference from APC-related familial adenomatous polyposis, because it relocates the genetic risk in a family from the offspring of an affected person to their siblings, and because a family history may appear negative across generations.
Autosomal recessive inheritance Penetrance: INCOMPLETE Expressivity: VARIABLE
Show evidence (4 references)
PMID:12606733 SUPPORT Human Clinical
"Germ-line MYH mutations predispose persons to a recessive phenotype, multiple adenomas, or polyposis coli."
States the recessive inheritance and the range of colorectal phenotypes it produces.
PMID:23035301 SUPPORT Other
"At conception, each sib of an affected individual has a 25% chance of being affected, a 50% chance of being a carrier with a small increased risk for CRC, and a 25% chance of being unaffected and not a carrier."
GeneReviews Genetic Counseling section, and the quantitative basis for this entry's claim that siblings rather than offspring are the high-risk relatives. Note the monoallelic-risk clause in this quote is contested by the case-control evidence below and is NOT curated here as established. Evidence source is OTHER because GeneReviews is an expert-authored review.
PMID:34981295 SUPPORT Human Clinical
"Using the largest colorectal and endometrial cancer cohorts and one of the largest breast cancer cohorts from a single case-control study, we did not observe a significant difference in the prevalence of monoallelic MUTYH PVs in these cohorts compared to controls."
PARTIAL rather than SUPPORT or REFUTE, because it cuts both ways against the item above. It strengthens the recessive architecture this block asserts — heterozygotes look like the general population — while contradicting that quote's clause about a small increased colorectal cancer risk in carriers. Scope limits that keep this from being decisive: individuals of European ancestry only, and panel-tested cases against gnomAD controls rather than a population cohort.
+ 1 more reference

Pathophysiology

4
Biallelic Germline MUTYH Base-Excision-Repair Deficiency
Both MUTYH alleles carry damaging germline variants. The two commonest founder alleles in European populations, Tyr165Cys and Gly382Asp, alter residues conserved in bacterial mutY — Tyr82, in the pseudo-helix-hairpin-helix motif predicted to determine mismatch specificity, and Gly253 — and the corresponding mutant proteins show significantly reduced adenine glycosylase activity against 8-oxoG:A and G:A substrates. Note this is a constitutional biallelic state present in every cell, not a first hit awaiting a second: MAP does not conform to the germline two-hit predisposition module.
MUTYH hgnc:7527 HUGO Gene Nomenclature Committee (hgnc) Relation: this pathophysiological event involves this gene This pathophysiological event involves decreased MUTYH (hgnc:7527). hgnc:7527 is a gene from the HUGO Gene Nomenclature Committee. ↓ DECREASED
Genetic context MUTYH hgnc:7527 HUGO Gene Nomenclature Committee (hgnc) Relation: this genetic context concerns this gene This genetic context concerns MUTYH (hgnc:7527). hgnc:7527 is a gene from the HUGO Gene Nomenclature Committee. variant_origin: GERMLINE allelic_hit_role: BIALLELIC_INACTIVATION allelic_event: MISSENSE_VARIANT allelic_event: PATHOGENIC_VARIANT zygosity: COMPOUND_HETEROZYGOUS functional_impact_category: LOSS_OF_FUNCTION
Constitutional biallelic MUTYH variants — homozygous or compound heterozygous, missense or protein-truncating — reducing adenine glycosylase activity.
adenine DNA glycosylase activity GO:0019104 Gene Ontology (GO) Relation: this pathophysiological event involves this molecular function This pathophysiological event involves decreased adenine DNA glycosylase activity, annotated with DNA N-glycosylase activity (GO:0019104). GO:0019104 is a molecular function from the Gene Ontology. ↓ DECREASED
Show evidence (2 references)
PMID:11818965 SUPPORT Human Clinical
"Analysis of the human homolog of mutY, MYH, showed that the siblings were compound heterozygotes for the nonconservative missense variants Tyr165Cys and Gly382Asp."
Identifies the biallelic germline genotype in the family that defined the syndrome.
PMID:11818965 SUPPORT In Vitro
"Assays of adenine glycosylase activity of the Tyr82Cys and Gly253Asp mutant proteins with 8-oxoG:A and G:A substrates show that their activity is reduced significantly."
Functional evidence that the founder alleles impair the enzyme's catalytic activity, establishing the repair deficiency at the protein level. Evidence source is IN_VITRO (biochemical glycosylase assays).
Unrepaired 8-Oxoguanine Mispairs and G:C to T:A Transversion Mutator Phenotype
The rate-limiting state: oxidative damage generates 8-oxoguanine, DNA polymerase misincorporates adenine opposite it, and the uncorrected mispair is fixed at the next replication as a G:C to T:A transversion. The result is a constitutional bias in the kind of mutation the cell accumulates rather than in the overall rate of chromosomal change, which is why MAP tumors carry a recognizable mutational signature. The parallel with bacteria is exact and was the reasoning that identified the gene: loss of mutM or mutY in Escherichia coli produces the same transversion excess.
base-excision repair GO:0006284 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves decreased base-excision repair (GO:0006284). GO:0006284 is a biological process from the Gene Ontology. ↓ DECREASED
Show evidence (1 reference)
PMID:11818965 SUPPORT Human Clinical
"Inherited defects of base excision repair have not been associated with any human genetic disorder, although mutations of the genes mutM and mutY, which function in Escherichia coli base excision repair, lead to increased transversions of G:C to T:A."
States the bacterial precedent that predicts the transversion signature, and establishes MAP as the first human disorder of inherited base-excision repair deficiency.
Somatic APC Inactivation by Transversion
The transversion-biased mutator phenotype inactivates APC somatically in colorectal epithelial cells, and the mutational signature is what betrays the mechanism: in the index family 15 of 18 somatic APC mutations across 11 tumors were of this type, a significantly greater proportion than in sporadic tumors or in APC-related familial adenomatous polyposis, and in confirmed biallelic carriers all somatic APC mutations found were G:C to T:A transversions. MAP therefore reaches the same effector lesion as classic FAP — APC loss and Wnt pathway activation — but by an entirely different route, with no germline APC involvement at all.
Genetic context APC hgnc:583 HUGO Gene Nomenclature Committee (hgnc) Relation: this genetic context concerns this gene This genetic context concerns APC (hgnc:583). hgnc:583 is a gene from the HUGO Gene Nomenclature Committee. variant_origin: SOMATIC allelic_event: PATHOGENIC_VARIANT functional_impact_category: LOSS_OF_FUNCTION
Somatic APC inactivation acquired through the MUTYH-deficient transversion signature, in a person with no germline APC mutation.
Show evidence (2 references)
PMID:11818965 SUPPORT Human Clinical
"Our findings link the inherited variants in MYH to the pattern of somatic APC mutation in family N and implicate defective base excision repair in predisposition to tumors in humans."
Establishes the causal link between the inherited repair defect and the somatic APC mutational pattern, which is the claim this node makes.
PMID:12606733 SUPPORT Human Clinical
"In the tumors of carriers of biallelic mutations, all somatic APC mutations were G:C-->T:A transversions."
Independent confirmation in a larger series that the somatic APC lesion in biallelic MUTYH carriers carries the predicted signature without exception.
Multiple Colorectal Adenomas and Carcinoma Risk
The clinical output: multiple colorectal adenomas, most often in the tens to low hundreds, with progression to colorectal carcinoma. The phenotype spans the attenuated and classic FAP ranges — about a third of patients with more than 15 adenomas carried biallelic MUTYH variants in the defining series — but severe polyposis with more than 1000 adenomas was not seen in biallelic carriers, so extreme polyp burden argues for APC rather than MUTYH. Extracolonic disease occurs in a minority.
Show evidence (2 references)
PMID:12606733 SUPPORT Human Clinical
"In the group with multiple adenomas, about one third of patients with more than 15 adenomas had biallelic MYH mutations."
Quantifies the diagnostic yield of MUTYH testing at the polyp-count threshold that defines the clinical presentation.
PMID:12606733 SUPPORT Human Clinical
"In the polyposis group, no patient with biallelic MYH mutations had severe disease (>1000 adenomas), but three had extracolonic disease."
Supports both the upper bound on polyp burden in MAP and the occurrence of extracolonic disease in a minority.

Pathograph

Use the checkboxes to hide or show graph categories. Hover nodes for evidence and cross-linked metadata.
Pathograph: causal mechanism network for MUTYH-Associated Polyposis Interactive directed graph showing how pathophysiology mechanisms, phenotypes, genetic factors and variants, experimental models, environmental triggers, and treatments relate through causal and linked edges.

Phenotypes

4
Digestive 1
Colorectal Carcinoma Colon cancer HP:0003003 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Colon cancer (HP:0003003). HP:0003003 is a phenotype from the Human Phenotype Ontology.
Show evidence (2 references)
PMID:11818965 SUPPORT Human Clinical
"We have studied family N, which is affected with multiple colorectal adenomas and carcinoma but lacks an inherited mutation of the adenomatous polyposis coli gene (APC) that is associated with familial adenomatous polyposis."
Establishes carcinoma as part of the phenotype in the index family, and the absence of germline APC that distinguishes MAP from FAP.
PMID:23035301 SUPPORT Other
"MUTYH polyposis (also referred to as MUTYH-associated polyposis, or MAP) is characterized by a greatly increased lifetime risk of colorectal cancer (CRC)."
GeneReviews Clinical Characteristics section, stating colorectal cancer as the dominant lifetime risk of the syndrome.
Other 3
Adenomatous Colonic Polyposis HP:0005227 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Adenomatous colonic polyposis (HP:0005227). HP:0005227 is a phenotype from the Human Phenotype Ontology.
Show evidence (2 references)
PMID:12606733 SUPPORT Human Clinical
"Six patients with multiple adenomas and eight patients with polyposis had biallelic germline MYH variants."
Documents biallelic MUTYH variants across both the multiple-adenoma and polyposis presentations.
PMID:23035301 SUPPORT Other
"Although typically associated with ten to a few hundred colonic adenomatous polyps, CRC develops in some individuals in the absence of polyposis."
GeneReviews Clinical Characteristics section, giving the polyp-count range and the important caveat that colorectal cancer can arise with no polyposis at all — so a normal polyp count does not exclude the diagnosis.
Duodenal Polyposis HP:0004783 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Duodenal polyposis (HP:0004783). HP:0004783 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:23035301 SUPPORT Other
"Duodenal adenomas are common, with an increased risk of duodenal cancer."
GeneReviews Clinical Characteristics section, establishing duodenal adenomas as a common feature and the basis for upper-tract surveillance.
Extracolonic Malignancy Risk Neoplasm by anatomical site HP:0011793 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Neoplasm by anatomical site (HP:0011793). HP:0011793 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:23035301 SUPPORT Other
"The risk for malignancies of the ovary and bladder is also increased, and there is some evidence of an increased risk for breast and endometrial cancer."
GeneReviews Clinical Characteristics section. The graded wording is preserved — ovary and bladder are stated as increased, breast and endometrium only as "some evidence".
🧬

Genetic Associations

1
MUTYH
Gene: MUTYH hgnc:7527 HUGO Gene Nomenclature Committee (hgnc) Relation: this disease-associated gene is this gene This disease-associated gene is MUTYH (hgnc:7527). hgnc:7527 is a gene from the HUGO Gene Nomenclature Committee. relationship_type: CAUSATIVE
Show evidence (1 reference)
PMID:12606733 SUPPORT Human Clinical
"For patients with about 15 or more colorectal adenomas--especially if no germ-line APC mutation has been identified and the family history is compatible with recessive inheritance--genetic testing of MYH is indicated for diagnosis and calculation of the level of risk in relatives."
Defines the clinical criteria for MUTYH testing derived from the causal relationship between biallelic variants and the phenotype.
💊

Medical Actions

2
Colonoscopic Surveillance and Polypectomy
Action: Therapeutic ProcedureNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is Therapeutic Procedure (NCIT:C49236). NCIT:C49236 is a clinical intervention from the NCI Thesaurus. NCIT:C49236
Because MAP reaches carcinoma through the same adenoma-carcinoma sequence as the APC-related polyposes, the clinical care of biallelic MUTYH carriers is the same as for classic or attenuated familial adenomatous polyposis: endoscopic surveillance with polypectomy, escalating to colectomy when polyp burden becomes unmanageable.
Mechanism Target:
INHIBITS Multiple Colorectal Adenomas and Carcinoma Risk — Surveillance and polypectomy do not correct the constitutional repair defect or the mutator phenotype; they remove initiated adenomas before they progress, acting on the consequence node.
Show evidence (2 references)
PMID:12606733 SUPPORT Human Clinical
"Clinical care of patients with biallelic MYH mutations should be similar to that of patients with classic or attenuated familial adenomatous polyposis."
The source's own recommendation that management follow the FAP model, which is what this treatment entry curates.
PMID:23035301 SUPPORT Other
"Colonoscopy with polypectomy every one to two years beginning at age 25-30 years; upper endoscopy and side viewing duodenoscopy every three months to four years beginning at age 30-35 years with subsequent follow up based on initial findings."
GeneReviews Management section, giving the concrete surveillance intervals and starting ages for both the colonic and duodenal arms.
Colectomy for Unmanageable Polyp Burden
Action: Surgical ProcedureNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is Surgical Procedure (NCIT:C15329). NCIT:C15329 is a clinical intervention from the NCI Thesaurus. NCIT:C15329
Polypectomy is continued until the size and density of polyps outstrip what endoscopy can manage, at which point subtotal colectomy or proctocolectomy is performed. The threshold is operational rather than a fixed polyp count.
Mechanism Target:
INHIBITS Multiple Colorectal Adenomas and Carcinoma Risk — Colectomy removes the at-risk colonic epithelium once endoscopic clearance is no longer feasible; it does not correct the constitutional repair defect, so duodenal surveillance continues afterwards.
Show evidence (1 reference)
PMID:23035301 SUPPORT Other
"Suspicious polyps identified on colonoscopy should be removed until polypectomy alone cannot manage the large size and density of the polyps, at which point either subtotal colectomy or proctocolectomy is performed."
GeneReviews Management section, stating both the escalation trigger and the two operations, which is what this treatment entry curates.
🔬

Diagnosis

1
Molecular Genetic Testing for Biallelic MUTYH Variants
The diagnosis requires demonstrating pathogenic variants in BOTH MUTYH alleles. This is the point at which MAP separates operationally from the dominant polyposis syndromes: a single variant does not establish the diagnosis, and the testing question in a polyposis patient with no germline APC variant and a recessive-compatible family history is specifically whether both MUTYH alleles are affected.
Genetic Testing NCIT:C15709 NCI Thesaurus (NCIT)
Results: Identification of biallelic germline pathogenic MUTYH variants establishes the diagnosis and makes sibling testing the priority for the family.
Show evidence (2 references)
PMID:23035301 SUPPORT Other
"The diagnosis is established in a proband by identification of biallelic germline pathogenic variants in MUTYH on molecular genetic testing."
GeneReviews Diagnosis/Testing section, stating the confirmatory test and the biallelic requirement that defines this disorder.
PMID:12606733 SUPPORT Human Clinical
"For patients with about 15 or more colorectal adenomas--especially if no germ-line APC mutation has been identified and the family history is compatible with recessive inheritance--genetic testing of MYH is indicated for diagnosis and calculation of the level of risk in relatives."
Gives the clinical threshold at which MUTYH testing is indicated, which is the selection criterion for the test curated here.
📊

Prevalence

1
Individuals of European ancestry
Carrier Frequency 1500.0 per 100,000 (1000.0–2000.0) >1 in 1,000
Monoallelic MUTYH carrier frequency of 1-2%, normalised to 1000-2000 per 100,000. This is the CARRIER frequency, not the prevalence of MUTYH-associated polyposis: the disorder is recessive, so affected individuals are the far rarer biallelic subset. The two must not be conflated when reading this record.
Show evidence (1 reference)
PMID:34981295 SUPPORT Human Clinical
"Additionally, frequencies among cancer cohorts were consistent with the published MUTYH carrier frequency of 1-2%."
Gives the monoallelic carrier frequency and corroborates it against the study's own cancer cohorts.
🔬

Clinical Trials

1
NCT07461246 NOT_APPLICABLE ACTIVE_NOT_RECRUITING
Italian national multicenter polyposis registry that enrolls MUTYH-associated polyposis alongside APC-related FAP, collecting the genotype-phenotype and surveillance-outcome data this entry cites as absent from single-center series.
Target Phenotypes: Adenomatous colonic polyposis HP:0005227 Human Phenotype Ontology (HP) Relation: this clinical trial targets this phenotype This clinical trial targets Adenomatous colonic polyposis (HP:0005227). HP:0005227 is a phenotype from the Human Phenotype Ontology. Colorectal carcinoma HP:0003003 Human Phenotype Ontology (HP) Relation: this clinical trial targets this phenotype This clinical trial targets Colorectal carcinoma, annotated with Colon cancer (HP:0003003). HP:0003003 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
clinicaltrials:NCT07461246 SUPPORT Human Clinical
"The registry includes patients with APC-related FAP (classic and attenuated forms), MUTYH-associated polyposis (MAP), and adenomatous polyposis not associated with APC or MUTYH mutations (NAMP)"
Registry record confirming MUTYH-associated polyposis is an enrolled cohort, so the registry's outcome data will be MAP-specific.
{ }

Source YAML

click to show
name: MUTYH-Associated Polyposis
creation_date: "2026-08-23T00:00:00Z"
category: Mendelian
categories:
- Hereditary Cancer Syndrome
- Cancer Predisposition Syndrome
- Gastrointestinal Neoplasia
parents:
- hereditary cancer-predisposing syndrome
disease_term:
  preferred_term: familial adenomatous polyposis 2
  term:
    id: MONDO:0012041
    label: familial adenomatous polyposis 2
synonyms:
- MAP
- MUTYH-associated polyposis
- MYH-associated polyposis
- autosomal recessive colorectal adenomatous polyposis
description: >-
  MUTYH-associated polyposis (MAP) is an autosomal recessive colorectal
  adenomatous polyposis caused by biallelic germline variants in MUTYH, the human
  homolog of bacterial mutY, which encodes the adenine DNA glycosylase of the
  base-excision-repair pathway. It is the exception that clarifies the rule among
  the polyposis syndromes, and its mechanism is deliberately NOT the Knudson
  two-hit route. MAP carriers inherit two damaged alleles of a repair gene rather
  than one damaged allele of a tumor suppressor; the resulting defect is a
  constitutional mutator phenotype rather than a constitutional loss of growth
  restraint. MUTYH excises adenine that DNA polymerase has misincorporated
  opposite 8-oxoguanine, the commonest oxidative base lesion. Without it, those
  mispairs are fixed as G:C to T:A transversions, and the tumor suppressor that
  actually initiates the adenoma — APC — is then inactivated somatically by
  exactly that mutational signature. The evidence for this chain is unusually
  direct: MUTYH was identified by noticing that a family with multiple colorectal
  adenomas but no germline APC mutation had tumors whose somatic APC mutations
  were overwhelmingly G:C to T:A transversions, and in confirmed biallelic
  carriers every somatic APC mutation found was of that type.
  Clinically MAP presents as multiple (typically 15 to a few hundred) colorectal
  adenomas, overlapping both attenuated and classic familial adenomatous
  polyposis, and its recognition matters because the recessive inheritance
  changes who in the family is at risk: siblings, not offspring, are the
  high-risk relatives.
inheritance:
- name: Autosomal recessive
  inheritance_term:
    preferred_term: Autosomal recessive inheritance
    term:
      id: HP:0000007
      label: Autosomal recessive inheritance
  penetrance: INCOMPLETE
  expressivity: VARIABLE
  description: >-
    MAP is inherited as an autosomal recessive trait; both MUTYH alleles must be
    damaged. This is the single most important practical difference from
    APC-related familial adenomatous polyposis, because it relocates the genetic
    risk in a family from the offspring of an affected person to their siblings,
    and because a family history may appear negative across generations.
  evidence:
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Germ-line MYH mutations predispose persons to a recessive phenotype,
      multiple adenomas, or polyposis coli.
    explanation: >-
      States the recessive inheritance and the range of colorectal phenotypes it
      produces.
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      At conception, each sib of an affected individual has a 25% chance of
      being affected, a 50% chance of being a carrier with a small increased
      risk for CRC, and a 25% chance of being unaffected and not a carrier.
    explanation: >-
      GeneReviews Genetic Counseling section, and the quantitative basis for
      this entry's claim that siblings rather than offspring are the high-risk
      relatives. Note the monoallelic-risk clause in this quote is contested by
      the case-control evidence below and is NOT curated here as established.
      Evidence source is OTHER because GeneReviews is an expert-authored review.
  - reference: PMID:34981295
    reference_title: "Monoallelic MUTYH pathogenic variants ascertained via multi-gene hereditary cancer panels are not associated with colorectal, endometrial, or breast cancer."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Using the largest colorectal and endometrial cancer cohorts and one of the
      largest breast cancer cohorts from a single case-control study, we did not
      observe a significant difference in the prevalence of monoallelic MUTYH
      PVs in these cohorts compared to controls.
    explanation: >-
      PARTIAL rather than SUPPORT or REFUTE, because it cuts both ways against
      the item above. It strengthens the recessive architecture this block
      asserts — heterozygotes look like the general population — while
      contradicting that quote's clause about a small increased colorectal
      cancer risk in carriers. Scope limits that keep this from being decisive:
      individuals of European ancestry only, and panel-tested cases against
      gnomAD controls rather than a population cohort.
  - reference: PMID:34981295
    reference_title: "Monoallelic MUTYH pathogenic variants ascertained via multi-gene hereditary cancer panels are not associated with colorectal, endometrial, or breast cancer."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      These findings suggest there is no association between colorectal,
      endometrial, or breast cancer and MUTYH heterozygosity in individuals of
      European ancestry.
    explanation: >-
      The authors' own conclusion, quoted with its ancestry restriction intact.
      Curators should not generalise it beyond European ancestry, and should not
      read it as bearing on BIALLELIC carriers, who are the subject of this
      entry.
pathophysiology:
- name: Biallelic Germline MUTYH Base-Excision-Repair Deficiency
  conforms_to: "genome_instability_mutation#Genome-Maintenance Defect or Replication Stress"
  description: >-
    Both MUTYH alleles carry damaging germline variants. The two commonest
    founder alleles in European populations, Tyr165Cys and Gly382Asp, alter
    residues conserved in bacterial mutY — Tyr82, in the
    pseudo-helix-hairpin-helix motif predicted to determine mismatch specificity, and Gly253 — and
    the corresponding mutant proteins show significantly reduced adenine
    glycosylase activity against 8-oxoG:A and G:A substrates. Note this is a
    constitutional biallelic state present in every cell, not a first hit
    awaiting a second: MAP does not conform to the germline two-hit
    predisposition module.
  role: trigger
  biological_scale: MOLECULAR
  gene:
    preferred_term: MUTYH
    modifier: DECREASED
    term:
      id: hgnc:7527
      label: MUTYH
  genetic_context:
    gene:
      preferred_term: MUTYH
      term:
        id: hgnc:7527
        label: MUTYH
    variant_origin: GERMLINE
    allelic_hit_role: BIALLELIC_INACTIVATION
    allelic_events:
    - MISSENSE_VARIANT
    - PATHOGENIC_VARIANT
    zygosity: COMPOUND_HETEROZYGOUS
    functional_impact_category: LOSS_OF_FUNCTION
    description: >-
      Constitutional biallelic MUTYH variants — homozygous or compound
      heterozygous, missense or protein-truncating — reducing adenine glycosylase
      activity.
  molecular_functions:
  - preferred_term: adenine DNA glycosylase activity
    term:
      id: GO:0019104
      label: DNA N-glycosylase activity
    modifier: DECREASED
  evidence:
  - reference: PMID:11818965
    reference_title: "Inherited variants of MYH associated with somatic G:C-->T:A mutations in colorectal tumors."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Analysis of the human homolog of mutY, MYH, showed that the siblings were
      compound heterozygotes for the nonconservative missense variants Tyr165Cys
      and Gly382Asp.
    explanation: >-
      Identifies the biallelic germline genotype in the family that defined the
      syndrome.
  - reference: PMID:11818965
    reference_title: "Inherited variants of MYH associated with somatic G:C-->T:A mutations in colorectal tumors."
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: >-
      Assays of adenine glycosylase activity of the Tyr82Cys and Gly253Asp
      mutant proteins with 8-oxoG:A and G:A substrates show that their activity
      is reduced significantly.
    explanation: >-
      Functional evidence that the founder alleles impair the enzyme's catalytic
      activity, establishing the repair deficiency at the protein level.
      Evidence source is IN_VITRO (biochemical glycosylase assays).
  downstream:
  - target: Unrepaired 8-Oxoguanine Mispairs and G:C to T:A Transversion Mutator Phenotype
    description: >-
      Without adenine glycosylase activity, adenine misincorporated opposite
      8-oxoguanine is not excised and the mispair is fixed at replication.

- name: Unrepaired 8-Oxoguanine Mispairs and G:C to T:A Transversion Mutator Phenotype
  conforms_to: "genome_instability_mutation#Mutator Phenotype and Chromosomal Instability"
  description: >-
    The rate-limiting state: oxidative damage generates 8-oxoguanine, DNA
    polymerase misincorporates adenine opposite it, and the uncorrected mispair
    is fixed at the next replication as a G:C to T:A transversion. The result is
    a constitutional bias in the kind of mutation the cell accumulates rather
    than in the overall rate of chromosomal change, which is why MAP tumors carry
    a recognizable mutational signature. The parallel with bacteria is exact and
    was the reasoning that identified the gene: loss of mutM or mutY in
    Escherichia coli produces the same transversion excess.
  role: central_effector
  biological_scale: MOLECULAR
  biological_processes:
  - preferred_term: base-excision repair
    term:
      id: GO:0006284
      label: base-excision repair
    modifier: DECREASED
  evidence:
  - reference: PMID:11818965
    reference_title: "Inherited variants of MYH associated with somatic G:C-->T:A mutations in colorectal tumors."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Inherited defects of base excision repair have not been associated with
      any human genetic disorder, although mutations of the genes mutM and mutY,
      which function in Escherichia coli base excision repair, lead to increased
      transversions of G:C to T:A.
    explanation: >-
      States the bacterial precedent that predicts the transversion signature,
      and establishes MAP as the first human disorder of inherited base-excision
      repair deficiency.
  downstream:
  - target: Somatic APC Inactivation by Transversion
    description: >-
      The transversion bias is applied genome-wide, but its consequence in
      colorectal epithelium is inactivation of APC.

- name: Somatic APC Inactivation by Transversion
  description: >-
    The transversion-biased mutator phenotype inactivates APC somatically in
    colorectal epithelial cells, and the mutational signature is what betrays the
    mechanism: in the index family 15 of 18 somatic APC mutations across 11
    tumors were of this type, a significantly greater proportion than in sporadic
    tumors or in APC-related familial adenomatous polyposis, and in confirmed
    biallelic carriers all somatic APC mutations found were G:C to T:A
    transversions. MAP therefore reaches the same effector lesion as classic FAP
    — APC loss and Wnt pathway activation — but by an entirely different route,
    with no germline APC involvement at all.
  role: effector
  biological_scale: MOLECULAR
  genetic_context:
    gene:
      preferred_term: APC
      term:
        id: hgnc:583
        label: APC
    variant_origin: SOMATIC
    allelic_events:
    - PATHOGENIC_VARIANT
    functional_impact_category: LOSS_OF_FUNCTION
    description: >-
      Somatic APC inactivation acquired through the MUTYH-deficient transversion
      signature, in a person with no germline APC mutation.
  evidence:
  - reference: PMID:11818965
    reference_title: "Inherited variants of MYH associated with somatic G:C-->T:A mutations in colorectal tumors."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Our findings link the inherited variants in MYH to the pattern of somatic
      APC mutation in family N and implicate defective base excision repair in
      predisposition to tumors in humans.
    explanation: >-
      Establishes the causal link between the inherited repair defect and the
      somatic APC mutational pattern, which is the claim this node makes.
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      In the tumors of carriers of biallelic mutations, all somatic APC
      mutations were G:C-->T:A transversions.
    explanation: >-
      Independent confirmation in a larger series that the somatic APC lesion in
      biallelic MUTYH carriers carries the predicted signature without exception.
  downstream:
  - target: Multiple Colorectal Adenomas and Carcinoma Risk
    description: >-
      APC-inactivated crypts initiate adenomas, which accumulate in numbers
      proportional to the constitutional mutation rate.

- name: Multiple Colorectal Adenomas and Carcinoma Risk
  description: >-
    The clinical output: multiple colorectal adenomas, most often in the tens to
    low hundreds, with progression to colorectal carcinoma. The phenotype spans
    the attenuated and classic FAP ranges — about a third of patients with more
    than 15 adenomas carried biallelic MUTYH variants in the defining series —
    but severe polyposis with more than 1000 adenomas was not seen in biallelic
    carriers, so extreme polyp burden argues for APC rather than MUTYH.
    Extracolonic disease occurs in a minority.
  role: consequence
  biological_scale: ORGANISM
  evidence:
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      In the group with multiple adenomas, about one third of patients with more
      than 15 adenomas had biallelic MYH mutations.
    explanation: >-
      Quantifies the diagnostic yield of MUTYH testing at the polyp-count
      threshold that defines the clinical presentation.
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      In the polyposis group, no patient with biallelic MYH mutations had severe
      disease (>1000 adenomas), but three had extracolonic disease.
    explanation: >-
      Supports both the upper bound on polyp burden in MAP and the occurrence of
      extracolonic disease in a minority.
phenotypes:
- category: Gastrointestinal
  name: Adenomatous Colonic Polyposis
  description: >-
    Multiple colorectal adenomas, typically 15 to several hundred, spanning the
    attenuated and classic polyposis ranges.
  phenotype_term:
    preferred_term: Adenomatous colonic polyposis
    term:
      id: HP:0005227
      label: Adenomatous colonic polyposis
  evidence:
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Six patients with multiple adenomas and eight patients with polyposis had
      biallelic germline MYH variants.
    explanation: >-
      Documents biallelic MUTYH variants across both the multiple-adenoma and
      polyposis presentations.
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Although typically associated with ten to a few hundred colonic
      adenomatous polyps, CRC develops in some individuals in the absence of
      polyposis.
    explanation: >-
      GeneReviews Clinical Characteristics section, giving the polyp-count range
      and the important caveat that colorectal cancer can arise with no
      polyposis at all — so a normal polyp count does not exclude the diagnosis.
- category: Gastrointestinal
  name: Colorectal Carcinoma
  description: >-
    Colorectal cancer arising through the adenoma-carcinoma sequence in the
    setting of a transversion-biased mutator phenotype.
  phenotype_term:
    preferred_term: Colon cancer
    term:
      id: HP:0003003
      label: Colon cancer
  evidence:
  - reference: PMID:11818965
    reference_title: "Inherited variants of MYH associated with somatic G:C-->T:A mutations in colorectal tumors."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      We have studied family N, which is affected with multiple colorectal
      adenomas and carcinoma but lacks an inherited mutation of the adenomatous
      polyposis coli gene (APC) that is associated with familial adenomatous
      polyposis.
    explanation: >-
      Establishes carcinoma as part of the phenotype in the index family, and
      the absence of germline APC that distinguishes MAP from FAP.
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      MUTYH polyposis (also referred to as MUTYH-associated polyposis, or MAP)
      is characterized by a greatly increased lifetime risk of colorectal cancer
      (CRC).
    explanation: >-
      GeneReviews Clinical Characteristics section, stating colorectal cancer as
      the dominant lifetime risk of the syndrome.
- category: Gastrointestinal
  name: Duodenal Polyposis
  description: >-
    Duodenal adenomas are a common extracolonic feature and carry their own
    cancer risk, which is why upper endoscopy with side-viewing duodenoscopy is
    part of surveillance rather than an optional extra.
  phenotype_term:
    preferred_term: Duodenal polyposis
    term:
      id: HP:0004783
      label: Duodenal polyposis
  evidence:
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Duodenal adenomas are common, with an increased risk of duodenal cancer.
    explanation: >-
      GeneReviews Clinical Characteristics section, establishing duodenal
      adenomas as a common feature and the basis for upper-tract surveillance.
- category: Neoplastic
  name: Extracolonic Malignancy Risk
  description: >-
    Beyond the gastrointestinal tract, risk is increased for ovarian and bladder
    malignancy, with weaker evidence for breast and endometrial cancer. These
    are recorded as an increased risk rather than as penetrant features.
  phenotype_term:
    preferred_term: Neoplasm by anatomical site
    term:
      id: HP:0011793
      label: Neoplasm by anatomical site
  evidence:
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      The risk for malignancies of the ovary and bladder is also increased, and
      there is some evidence of an increased risk for breast and endometrial
      cancer.
    explanation: >-
      GeneReviews Clinical Characteristics section. The graded wording is
      preserved — ovary and bladder are stated as increased, breast and
      endometrium only as "some evidence".
genetic:
- name: MUTYH
  gene_term:
    preferred_term: MUTYH
    term:
      id: hgnc:7527
      label: MUTYH
  relationship_type: CAUSATIVE
  notes: >-
    MUTYH encodes the adenine DNA glycosylase of base-excision repair. Biallelic
    germline variants cause MUTYH-associated polyposis; monoallelic carriage
    confers at most a modest colorectal cancer risk and is not this disorder.
  evidence:
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      For patients with about 15 or more colorectal adenomas--especially if no
      germ-line APC mutation has been identified and the family history is
      compatible with recessive inheritance--genetic testing of MYH is indicated
      for diagnosis and calculation of the level of risk in relatives.
    explanation: >-
      Defines the clinical criteria for MUTYH testing derived from the causal
      relationship between biallelic variants and the phenotype.
treatments:
- name: Colonoscopic Surveillance and Polypectomy
  notes: >-
    Curated under `treatments:` as a management intervention, not as a claim
    that endoscopy is itself therapeutic. The initiating lesion here is
    constitutional and cannot be reversed — in MAP that is biallelic MUTYH
    base-excision-repair failure, not a two-hit tumor suppressor architecture
    (see the entry-level `notes:` on the boundary with FAP) — so the only
    available lever is stage at detection: surveillance is the intervention
    that converts an otherwise unavoidable carcinoma into a resectable polyp.
    Where a `target_mechanisms` link is present it points at the
    clinical-outcome node for that reason, and never at an upstream
    mechanistic node.
  description: >-
    Because MAP reaches carcinoma through the same adenoma-carcinoma sequence as
    the APC-related polyposes, the clinical care of biallelic MUTYH carriers is
    the same as for classic or attenuated familial adenomatous polyposis:
    endoscopic surveillance with polypectomy, escalating to colectomy when polyp
    burden becomes unmanageable.
  therapeutic_modality: DEVICE
  treatment_term:
    preferred_term: Therapeutic Procedure
    term:
      id: NCIT:C49236
      label: Therapeutic Procedure
  target_mechanisms:
  - target: Multiple Colorectal Adenomas and Carcinoma Risk
    treatment_effect: INHIBITS
    description: >-
      Surveillance and polypectomy do not correct the constitutional repair
      defect or the mutator phenotype; they remove initiated adenomas before
      they progress, acting on the consequence node.
  evidence:
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Clinical care of patients with biallelic MYH mutations should be similar
      to that of patients with classic or attenuated familial adenomatous
      polyposis.
    explanation: >-
      The source's own recommendation that management follow the FAP model,
      which is what this treatment entry curates.
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Colonoscopy with polypectomy every one to two years beginning at age 25-30
      years; upper endoscopy and side viewing duodenoscopy every three months to
      four years beginning at age 30-35 years with subsequent follow up based on
      initial findings.
    explanation: >-
      GeneReviews Management section, giving the concrete surveillance intervals
      and starting ages for both the colonic and duodenal arms.
- name: Colectomy for Unmanageable Polyp Burden
  description: >-
    Polypectomy is continued until the size and density of polyps outstrip what
    endoscopy can manage, at which point subtotal colectomy or proctocolectomy
    is performed. The threshold is operational rather than a fixed polyp count.
  therapeutic_modality: SURGERY
  treatment_term:
    preferred_term: Surgical Procedure
    term:
      id: NCIT:C15329
      label: Surgical Procedure
  target_mechanisms:
  - target: Multiple Colorectal Adenomas and Carcinoma Risk
    treatment_effect: INHIBITS
    description: >-
      Colectomy removes the at-risk colonic epithelium once endoscopic clearance
      is no longer feasible; it does not correct the constitutional repair
      defect, so duodenal surveillance continues afterwards.
  evidence:
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Suspicious polyps identified on colonoscopy should be removed until
      polypectomy alone cannot manage the large size and density of the polyps,
      at which point either subtotal colectomy or proctocolectomy is performed.
    explanation: >-
      GeneReviews Management section, stating both the escalation trigger and
      the two operations, which is what this treatment entry curates.
diagnosis:
- name: Molecular Genetic Testing for Biallelic MUTYH Variants
  description: >-
    The diagnosis requires demonstrating pathogenic variants in BOTH MUTYH
    alleles. This is the point at which MAP separates operationally from the
    dominant polyposis syndromes: a single variant does not establish the
    diagnosis, and the testing question in a polyposis patient with no germline
    APC variant and a recessive-compatible family history is specifically
    whether both MUTYH alleles are affected.
  diagnosis_term:
    preferred_term: Genetic Testing
    term:
      id: NCIT:C15709
      label: Genetic Testing
  results: >-
    Identification of biallelic germline pathogenic MUTYH variants establishes
    the diagnosis and makes sibling testing the priority for the family.
  evidence:
  - reference: PMID:23035301
    reference_title: "MUTYH Polyposis."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      The diagnosis is established in a proband by identification of biallelic
      germline pathogenic variants in MUTYH on molecular genetic testing.
    explanation: >-
      GeneReviews Diagnosis/Testing section, stating the confirmatory test and
      the biallelic requirement that defines this disorder.
  - reference: PMID:12606733
    reference_title: "Multiple colorectal adenomas, classic adenomatous polyposis, and germ-line mutations in MYH."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      For patients with about 15 or more colorectal adenomas--especially if no
      germ-line APC mutation has been identified and the family history is
      compatible with recessive inheritance--genetic testing of MYH is indicated
      for diagnosis and calculation of the level of risk in relatives.
    explanation: >-
      Gives the clinical threshold at which MUTYH testing is indicated, which is
      the selection criterion for the test curated here.
references:
- reference: PMID:23035301
  title: "MUTYH Polyposis."
  tags:
  - GeneReviews
prevalence:
- population: Individuals of European ancestry
  measure_type: CARRIER_FREQUENCY
  prevalence_class: ABOVE_1_IN_1000
  rate_per_100000: 1500.0
  rate_low: 1000.0
  rate_high: 2000.0
  notes: >-
    Monoallelic MUTYH carrier frequency of 1-2%, normalised to 1000-2000 per
    100,000. This is the CARRIER frequency, not the prevalence of
    MUTYH-associated polyposis: the disorder is recessive, so affected
    individuals are the far rarer biallelic subset. The two must not be
    conflated when reading this record.
  evidence:
  - reference: PMID:34981295
    reference_title: "Monoallelic MUTYH pathogenic variants ascertained via multi-gene hereditary cancer panels are not associated with colorectal, endometrial, or breast cancer."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Additionally, frequencies among cancer cohorts were consistent with the
      published MUTYH carrier frequency of 1-2%.
    explanation: >-
      Gives the monoallelic carrier frequency and corroborates it against the
      study's own cancer cohorts.
clinical_trials:
- name: NCT07461246
  phase: NOT_APPLICABLE
  status: ACTIVE_NOT_RECRUITING
  description: >-
    Italian national multicenter polyposis registry that enrolls
    MUTYH-associated polyposis alongside APC-related FAP, collecting the
    genotype-phenotype and surveillance-outcome data this entry cites as
    absent from single-center series.
  target_phenotypes:
  - preferred_term: Adenomatous colonic polyposis
    term:
      id: HP:0005227
      label: Adenomatous colonic polyposis
  - preferred_term: Colorectal carcinoma
    term:
      id: HP:0003003
      label: Colon cancer
  evidence:
  - reference: clinicaltrials:NCT07461246
    reference_title: Rete Italiana Poliposi Adenomatosa Familiare (RIPAF)
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      The registry includes patients with APC-related FAP (classic and attenuated forms), MUTYH-associated polyposis (MAP), and adenomatous polyposis not associated with APC or MUTYH mutations (NAMP)
    explanation: >-
      Registry record confirming MUTYH-associated polyposis is an enrolled
      cohort, so the registry's outcome data will be MAP-specific.
notes: >-
  Boundary with familial adenomatous polyposis. MUTYH-associated polyposis and
  APC-related FAP are kept as separate entries because they are different
  mechanisms that converge on a similar colonic phenotype, not one disease with
  two genes. FAP is an autosomal dominant two-hit tumor suppressor syndrome and
  conforms to `germline_two_hit_tumor_predisposition`; MAP is autosomal
  recessive, requires biallelic MUTYH from the outset, and acts through failed
  base excision repair of 8-oxoguanine, so it conforms to
  `genome_instability_mutation` instead. The practical consequences differ too:
  MAP siblings carry a one-in-four recurrence risk while offspring are usually
  unaffected, which is the inverse of the FAP counselling picture. A shared
  polyposis registry may enrol both, and this entry cites one that does, but
  that is cohort convenience rather than a claim of shared mechanism.
📚

References & Deep Research

References

1
MUTYH Polyposis.
No top-level findings curated for this source.