Dilated Cardiomyopathy 1G

Genetic MONDO:0011400 Pathograph 5 Show in embeddings browser Dilated Cardiomyopathy Genetic Disorder

Dilated cardiomyopathy 1G (CMD1G) is the TTN-related form of familial isolated dilated cardiomyopathy and the single commonest genetic cause of the disease. TTN encodes titin, the largest protein in the human proteome: a single molecule spans half the sarcomere from Z-disc to M-band, acting as a molecular ruler for sarcomere assembly, as the elastic spring that sets passive myocardial stiffness, and as a mechanosensing scaffold. The disease alleles are titin truncating variants (TTNtv) - nonsense, frameshift, splice-disrupting, and copy-number changes predicted to truncate the full-length polypeptide - which Herman and colleagues established in 2012 as a common cause of dilated cardiomyopathy, present in roughly a quarter of familial and a fifth of sporadic cases. Position within the gene is the critical determinant of pathogenicity: truncations in the inextensible A-band, the region that binds myosin and myosin-binding protein C, are the disease-associated class, whereas TTNtv elsewhere - and TTNtv found at appreciable frequency in the general population - are largely benign, so a truncating variant is not by itself a diagnosis. Inheritance is autosomal dominant with high penetrance in clinically ascertained families but substantially lower penetrance among population carriers. TTNtv-positive DCM is clinically distinctive for an appreciable burden of atrial and ventricular arrhythmia alongside the ventricular dilation and systolic dysfunction.

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1
Inheritance
4
Pathophys.
3
Phenotypes
5
Pathograph
1
Genes
3
Medical Actions
1
References
🏷

Classifications

Harrison's Part
CARDIOVASCULAR GENETICS ENVIRONMENT DISEASE
👪

Inheritance

1
Autosomal dominant HP:0000006
CMD1G is inherited as an autosomal dominant trait; a single TTN truncating allele causes disease. Penetrance is high in clinically ascertained families - exceeding 95% among family members over 40 years of age in the founding study - but is substantially lower among TTNtv carriers ascertained from the general population, so a family-based penetrance figure must not be quoted to an incidentally genotyped individual.
Autosomal dominant inheritance Penetrance: INCOMPLETE
Show evidence (2 references)
"TTN | HGNC:12403 | dilated cardiomyopathy | MONDO:0005021 | AD | Definitive"
ClinGen's Dilated Cardiomyopathy Gene Curation Expert Panel classifies the TTN-dilated cardiomyopathy relationship as Definitive with autosomal dominant inheritance.
PMID:22335739 SUPPORT Human Clinical
"the penetrance of TTN truncating mutations was more than 95%"
Quantifies penetrance among family members over 40 years of age in the founding TTN dilated cardiomyopathy study - the clinically ascertained figure, higher than in population carriers.

Pathophysiology

4
TTN Truncating Variant in the A-Band
The initiating lesion in CMD1G is a titin truncating variant (TTNtv) - a nonsense, frameshift, splice-disrupting, or copy-number change predicted to substantially alter the full-length titin polypeptide. Position is the critical determinant of pathogenicity: disease-associated TTNtv are located predominantly in the inextensible A-band, the segment that binds myosin and myosin-binding protein C and is thought to be critical for biomechanical sensing and signaling. This positional dependence is what separates pathogenic from benign truncations, since TTNtv also occur in the general population.
Cardiomyocyte CL:0000746 Cell Ontology (CL) Relation: this pathophysiological event involves this cell type This pathophysiological event involves Cardiomyocyte, annotated with cardiac muscle cell (CL:0000746). CL:0000746 is a cell type from the Cell Ontology.
TTN hgnc:12403 HUGO Gene Nomenclature Committee (hgnc) Relation: this pathophysiological event involves this gene This pathophysiological event involves TTN (hgnc:12403). hgnc:12403 is a gene from the HUGO Gene Nomenclature Committee.
Sarcomere Organization GO:0045214 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves abnormal Sarcomere Organization (GO:0045214). GO:0045214 is a biological process from the Gene Ontology. ⚠ ABNORMAL
Sarcomeric A band GO:0031672 Gene Ontology (GO) Relation: this pathophysiological event involves this cellular component This pathophysiological event involves Sarcomeric A band, annotated with A band (GO:0031672). GO:0031672 is a cellular component from the Gene Ontology.
Show evidence (3 references)
PMID:22335739 SUPPORT Human Clinical
"We prioritized for analysis four types of variants - i.e., nonsense, frameshift, splicing, and copynumber - that (unlike most missense variants) are predicted to have a profound effect on the structure of full-length titin polypeptides"
Defines the TTN truncating variant class that constitutes the disease allele in this entity.
PMID:25589632 SUPPORT Human Clinical
"The inextensible A-band binds myosin and myosin-binding protein and is thought to be critical for biomechanical sensing and signaling."
Establishes the functional significance of the A-band, the region in which pathogenic truncations are concentrated.
PMID:25589632 SUPPORT Human Clinical
"Our previous study of severe and familial DCM identified TTNtv that were located predominantly in the A-band"
Documents the A-band concentration of disease-associated TTNtv, the positional rule this node encodes.
Sarcomere Insufficiency and Impaired Mechanosensing
Titin is the molecular ruler and elastic backbone of the sarcomere: it templates myofibril assembly, provides the passive restoring force of diastole, and transduces mechanical strain into signalling at the Z-disc and M-band. A truncating allele removes full-length titin from half the molecular pool, and the truncated product does not substitute for it. The consequence is a sarcomere that assembles and transmits force imperfectly and that senses mechanical load abnormally - a contractile and mechanosensing deficit rather than the hypercontractility of the hypertrophic sarcomeropathies. Human heart tissue analyses integrating RNA and protein data underpin the discrimination of pathogenic from benign truncations on this basis.
Cardiomyocyte CL:0000746 Cell Ontology (CL) Relation: this pathophysiological event involves this cell type This pathophysiological event involves Cardiomyocyte, annotated with cardiac muscle cell (CL:0000746). CL:0000746 is a cell type from the Cell Ontology.
Cardiac Myofibril Assembly GO:0055003 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves abnormal Cardiac Myofibril Assembly (GO:0055003). GO:0055003 is a biological process from the Gene Ontology. ⚠ ABNORMAL Muscle Contraction GO:0006936 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves decreased Muscle Contraction (GO:0006936). GO:0006936 is a biological process from the Gene Ontology. ↓ DECREASED
Show evidence (2 references)
PMID:25589632 SUPPORT Human Clinical
"we coupled TTN gene sequencing with cardiac phenotyping in 5267 individuals across the spectrum of cardiac physiology and integrated these data with RNA and protein analyses of human heart tissues"
Describes the integrated human tissue RNA/protein analysis on which the molecular consequences of TTN truncation in this node rest.
PMID:25589632 SUPPORT Human Clinical
"A-band are excluded by alternate splicing, or whether the pathogenesis of TTN DCM is determined either by critical functional elements located in the A-band, or by the length of a truncated TTN protein product."
Records the open question this node deliberately does not over-resolve - whether pathogenicity follows from A-band functional elements or from truncated-product length. PARTIAL because the mechanism is stated as alternatives rather than settled.
Ventricular Dilation and Systolic Dysfunction
Chronic sarcomeric insufficiency produces the defining structural remodeling of dilated cardiomyopathy: progressive left ventricular chamber enlargement with wall thinning, myocyte loss, and interstitial fibrosis, accompanied by falling ejection fraction. Unlike the hypertrophic sarcomeropathies, the geometric response is dilation rather than wall thickening, and neurohormonal activation drives the remodeling forward.
Cardiomyocyte CL:0000746 Cell Ontology (CL) Relation: this pathophysiological event involves this cell type This pathophysiological event involves Cardiomyocyte, annotated with cardiac muscle cell (CL:0000746). CL:0000746 is a cell type from the Cell Ontology. Cardiac Fibroblast CL:0002548 Cell Ontology (CL) Relation: this pathophysiological event involves this cell type This pathophysiological event involves Cardiac Fibroblast, annotated with fibroblast of cardiac tissue (CL:0002548). CL:0002548 is a cell type from the Cell Ontology.
Heart Contraction GO:0060047 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves decreased Heart Contraction (GO:0060047). GO:0060047 is a biological process from the Gene Ontology. ↓ DECREASED Extracellular Matrix Organization GO:0030198 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves increased Extracellular Matrix Organization (GO:0030198). GO:0030198 is a biological process from the Gene Ontology. ↑ INCREASED
Left ventricle UBERON:0002084 Uberon multi-species anatomy ontology (UBERON) Relation: this pathophysiological event occurs in this anatomical location This pathophysiological event occurs in Left ventricle, annotated with heart left ventricle (UBERON:0002084). UBERON:0002084 is an anatomical location from the Uberon multi-species anatomy ontology.
Show evidence (1 reference)
PMID:25589632 SUPPORT Human Clinical
"TTNtv is the most common genetic cause of DCM in ambulant patients in the community, identify clinically important manifestations of TTNtv-positive DCM, and define the penetrance and outcomes of TTNtv in the general population."
Establishes TTNtv as the leading genetic cause of the dilated cardiomyopathy phenotype this node represents, in a community-ascertained cohort.
Heart Failure and Arrhythmic Risk
The dilated, poorly contracting, fibrotic ventricle produces the clinical endpoints of CMD1G: progressive systolic heart failure requiring standard heart-failure therapy and, in advanced disease, mechanical support or transplantation. TTNtv-positive DCM additionally carries a clinically important burden of atrial and ventricular arrhythmia, so rhythm surveillance is part of management rather than an incidental concern.
Cardiomyocyte CL:0000746 Cell Ontology (CL) Relation: this pathophysiological event involves this cell type This pathophysiological event involves Cardiomyocyte, annotated with cardiac muscle cell (CL:0000746). CL:0000746 is a cell type from the Cell Ontology.
Heart Contraction GO:0060047 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves abnormal Heart Contraction (GO:0060047). GO:0060047 is a biological process from the Gene Ontology. ⚠ ABNORMAL
Show evidence (1 reference)
PMID:25589632 SUPPORT Human Clinical
"identify clinically important manifestations of TTNtv-positive DCM, and define the penetrance and outcomes of TTNtv in the general population."
Anchors the clinically important manifestations and outcomes of TTNtv-positive dilated cardiomyopathy that constitute this endpoint node.

Pathograph

Use the checkboxes to hide or show graph categories. Hover nodes for evidence and cross-linked metadata.
Pathograph: causal mechanism network for Dilated Cardiomyopathy 1G 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

3
Dilated Cardiomyopathy OBLIGATE Cardiovascular HP:0001644 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Dilated cardiomyopathy (HP:0001644). HP:0001644 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:22335739 SUPPORT Human Clinical
"TTN truncating mutations are a common cause of dilated cardiomyopathy, occurring in approximately 25% of familial cases of idiopathic dilated cardiomyopathy and in 18% of sporadic cases."
Establishes dilated cardiomyopathy as the phenotype caused by TTN truncating variants.
Congestive Heart Failure Cardiovascular HP:0001635 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Congestive heart failure (HP:0001635). HP:0001635 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:21353195 SUPPORT Human Clinical
"Dilated cardiomyopathy commonly causes heart failure and is the most frequent precipitating cause of heart transplantation."
States the heart-failure and transplantation burden of dilated cardiomyopathy, the endpoint this phenotype records. Cited from a DCM gene-discovery paper that states the general clinical burden.
Ventricular Arrhythmia Cardiovascular HP:0004308 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Ventricular arrhythmia (HP:0004308). HP:0004308 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:25589632 SUPPORT Human Clinical
"identify clinically important manifestations of TTNtv-positive DCM, and define the penetrance and outcomes of TTNtv in the general population."
The community cohort defining the clinically important manifestations of TTNtv-positive DCM, among which arrhythmia is prominent; PARTIAL because the quoted sentence names the manifestations collectively rather than arrhythmia specifically.
🧬

Genetic Associations

1
TTN
Gene: TTN hgnc:12403 HUGO Gene Nomenclature Committee (hgnc) Relation: this disease-associated gene is this gene This disease-associated gene is TTN (hgnc:12403). hgnc:12403 is a gene from the HUGO Gene Nomenclature Committee. relationship_type: CAUSATIVE
Show evidence (2 references)
"TTN | HGNC:12403 | dilated cardiomyopathy | MONDO:0005021 | AD | Definitive"
ClinGen's Gene Curation Expert Panel classifies this gene-disease relationship as Definitive, which is the authority for curating the gene as CAUSATIVE rather than a candidate.
PMID:22335739 SUPPORT Human Clinical
"the penetrance of TTN truncating mutations was more than 95%"
Gene-level penetrance, quoted with the caveat that matters clinically: this is the figure among family members over 40 in clinically ascertained families, and penetrance among TTNtv carriers ascertained from the general population is substantially lower. PARTIAL because the number cannot be transferred to an incidentally genotyped individual.
💊

Medical Actions

3
Heart Failure Pharmacotherapy
Action: PharmacotherapyNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is Pharmacotherapy (NCIT:C15986). NCIT:C15986 is a clinical intervention from the NCI Thesaurus. NCIT:C15986
Agent: renin-angiotensin-aldosterone system inhibitor NCIT:C247 NCI Thesaurus (NCIT) Relation: this treatment uses this therapeutic agent This treatment uses renin-angiotensin-aldosterone system inhibitor, annotated with ACE Inhibitor (NCIT:C247). NCIT:C247 is a therapeutic agent from the NCI Thesaurus. beta-blocker NCIT:C29576 NCI Thesaurus (NCIT) Relation: this treatment uses this therapeutic agent This treatment uses beta-blocker, annotated with Beta-Adrenergic Antagonist (NCIT:C29576). NCIT:C29576 is a therapeutic agent from the NCI Thesaurus. mineralocorticoid receptor antagonist NCIT:C101255 NCI Thesaurus (NCIT) Relation: this treatment uses this therapeutic agent This treatment uses mineralocorticoid receptor antagonist, annotated with Aldosterone Receptor Antagonist (NCIT:C101255). NCIT:C101255 is a therapeutic agent from the NCI Thesaurus.
Standard guideline-directed heart-failure therapy (renin-angiotensin-aldosterone system inhibition, beta-blockade, mineralocorticoid receptor antagonism) targets the neurohormonal drive of adverse remodeling.
Implantable Cardioverter-Defibrillator
Action: implantable cardioverter-defibrillator placementNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is implantable cardioverter-defibrillator placement (NCIT:C80435). NCIT:C80435 is a clinical intervention from the NCI Thesaurus. Ontology label: Implantable Cardioverter-Defibrillator Placement NCIT:C80435
Considered for prevention of sudden cardiac death given the arrhythmic burden of TTNtv-positive dilated cardiomyopathy.
Heart Transplantation
Action: organ transplantationNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is organ transplantation (NCIT:C15289). NCIT:C15289 is a clinical intervention from the NCI Thesaurus. Ontology label: Organ Transplantation NCIT:C15289
Advanced, refractory disease is an indication for transplantation; dilated cardiomyopathy is the most frequent precipitating cause of heart transplant.
📊

Prevalence

1
Familial idiopathic dilated cardiomyopathy
Unknown Unknown
TTN truncating variants account for approximately 25% of familial and 18% of sporadic idiopathic dilated cardiomyopathy - a share-of-cases figure, not a population rate. This makes TTN the largest single genetic contributor to DCM.
Show evidence (1 reference)
PMID:22335739 SUPPORT Human Clinical
"TTN truncating mutations are a common cause of dilated cardiomyopathy, occurring in approximately 25% of familial cases of idiopathic dilated cardiomyopathy and in 18% of sporadic cases."
Gives the familial and sporadic share of dilated cardiomyopathy attributable to TTN truncating variants.
{ }

Source YAML

click to show
name: Dilated Cardiomyopathy 1G
creation_date: "2026-08-22T00:00:00Z"
synonyms:
- CMD1G
- TTN familial isolated dilated cardiomyopathy
- dilated cardiomyopathy type 1G
- cardiomyopathy, dilated, 1G
- titin-related dilated cardiomyopathy
- TTNtv dilated cardiomyopathy
description: >-
  Dilated cardiomyopathy 1G (CMD1G) is the TTN-related form of familial isolated
  dilated cardiomyopathy and the single commonest genetic cause of the disease.
  TTN encodes titin, the largest protein in the human proteome: a single molecule
  spans half the sarcomere from Z-disc to M-band, acting as a molecular ruler for
  sarcomere assembly, as the elastic spring that sets passive myocardial
  stiffness, and as a mechanosensing scaffold. The disease alleles are titin
  truncating variants (TTNtv) - nonsense, frameshift, splice-disrupting, and
  copy-number changes predicted to truncate the full-length polypeptide - which
  Herman and colleagues established in 2012 as a common cause of dilated
  cardiomyopathy, present in roughly a quarter of familial and a fifth of sporadic
  cases. Position within the gene is the critical determinant of pathogenicity:
  truncations in the inextensible A-band, the region that binds myosin and
  myosin-binding protein C, are the disease-associated class, whereas TTNtv
  elsewhere - and TTNtv found at appreciable frequency in the general population -
  are largely benign, so a truncating variant is not by itself a diagnosis.
  Inheritance is autosomal dominant with high penetrance in clinically ascertained
  families but substantially lower penetrance among population carriers.
  TTNtv-positive DCM is clinically distinctive for an appreciable burden of
  atrial and ventricular arrhythmia alongside the ventricular dilation and systolic
  dysfunction.
category: Genetic
classifications:
  harrisons_chapter:
  - classification_value: CARDIOVASCULAR
  - classification_value: GENETICS_ENVIRONMENT_DISEASE
disease_term:
  preferred_term: dilated cardiomyopathy 1G
  term:
    id: MONDO:0011400
    label: dilated cardiomyopathy 1G
parents:
- Dilated Cardiomyopathy
- Genetic Disorder
inheritance:
- name: Autosomal dominant
  description: >-
    CMD1G is inherited as an autosomal dominant trait; a single TTN truncating
    allele causes disease. Penetrance is high in clinically ascertained families -
    exceeding 95% among family members over 40 years of age in the founding study -
    but is substantially lower among TTNtv carriers ascertained from the general
    population, so a family-based penetrance figure must not be quoted to an
    incidentally genotyped individual.
  inheritance_term:
    preferred_term: Autosomal dominant inheritance
    term:
      id: HP:0000006
      label: Autosomal dominant inheritance
  penetrance: INCOMPLETE
  evidence:
  - reference: CGGV:assertion_1ec53217-814e-44b3-a7b7-0f18311c20f3-2025-05-30T160000.000Z
    reference_title: "TTN / dilated cardiomyopathy (Definitive)"
    supports: SUPPORT
    evidence_source: OTHER
    snippet: "TTN | HGNC:12403 | dilated cardiomyopathy | MONDO:0005021 | AD | Definitive"
    explanation: >-
      ClinGen's Dilated Cardiomyopathy Gene Curation Expert Panel classifies the
      TTN-dilated cardiomyopathy relationship as Definitive with autosomal
      dominant inheritance.
  - reference: PMID:22335739
    reference_title: "Truncations of titin causing dilated cardiomyopathy."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "the penetrance of TTN truncating mutations was more than 95%"
    explanation: >-
      Quantifies penetrance among family members over 40 years of age in the
      founding TTN dilated cardiomyopathy study - the clinically ascertained
      figure, higher than in population carriers.
prevalence:
- population: Familial idiopathic dilated cardiomyopathy
  measure_type: UNKNOWN
  prevalence_class: UNKNOWN
  notes: >-
    TTN truncating variants account for approximately 25% of familial and 18% of
    sporadic idiopathic dilated cardiomyopathy - a share-of-cases figure, not a
    population rate. This makes TTN the largest single genetic contributor to DCM.
  evidence:
  - reference: PMID:22335739
    reference_title: "Truncations of titin causing dilated cardiomyopathy."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      TTN truncating mutations are a common cause of dilated cardiomyopathy,
      occurring in approximately 25% of familial cases of idiopathic dilated
      cardiomyopathy and in 18% of sporadic cases.
    explanation: >-
      Gives the familial and sporadic share of dilated cardiomyopathy attributable
      to TTN truncating variants.
pathophysiology:
- name: TTN Truncating Variant in the A-Band
  biological_scale: MOLECULAR
  role: trigger
  conforms_to: "cardiomyopathy_maladaptive_remodeling#Primary Cardiomyocyte Insult"
  description: >-
    The initiating lesion in CMD1G is a titin truncating variant (TTNtv) -
    a nonsense, frameshift, splice-disrupting, or copy-number change predicted to
    substantially alter the full-length titin polypeptide. Position is the critical
    determinant of pathogenicity: disease-associated TTNtv are located
    predominantly in the inextensible A-band, the segment that binds myosin and
    myosin-binding protein C and is thought to be critical for biomechanical
    sensing and signaling. This positional dependence is what separates pathogenic
    from benign truncations, since TTNtv also occur in the general population.
  genes:
  - preferred_term: TTN
    term:
      id: hgnc:12403
      label: TTN
  cell_types:
  - preferred_term: Cardiomyocyte
    term:
      id: CL:0000746
      label: cardiac muscle cell
  biological_processes:
  - preferred_term: Sarcomere Organization
    term:
      id: GO:0045214
      label: sarcomere organization
    modifier: ABNORMAL
  cellular_components:
  - preferred_term: Sarcomeric A band
    term:
      id: GO:0031672
      label: A band
  evidence:
  - reference: PMID:22335739
    reference_title: "Truncations of titin causing dilated cardiomyopathy."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      We prioritized for analysis four types of variants - i.e., nonsense,
      frameshift, splicing, and copynumber - that (unlike most missense variants)
      are predicted to have a profound effect on the structure of full-length titin
      polypeptides
    explanation: >-
      Defines the TTN truncating variant class that constitutes the disease allele
      in this entity.
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      The inextensible A-band binds myosin and myosin-binding protein and is
      thought to be critical for biomechanical sensing and signaling.
    explanation: >-
      Establishes the functional significance of the A-band, the region in which
      pathogenic truncations are concentrated.
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "Our previous study of severe and familial DCM identified TTNtv that were located predominantly in the A-band"
    explanation: >-
      Documents the A-band concentration of disease-associated TTNtv, the
      positional rule this node encodes.
  downstream:
  - target: Sarcomere Insufficiency and Impaired Mechanosensing
    causal_link_type: DIRECT
    description: >-
      Loss of full-length titin from the sarcomere degrades assembly, elastic
      recoil, and mechanosensing.
- name: Sarcomere Insufficiency and Impaired Mechanosensing
  biological_scale: MOLECULAR
  role: effector
  description: >-
    Titin is the molecular ruler and elastic backbone of the sarcomere: it
    templates myofibril assembly, provides the passive restoring force of diastole,
    and transduces mechanical strain into signalling at the Z-disc and M-band. A
    truncating allele removes full-length titin from half the molecular pool, and
    the truncated product does not substitute for it. The consequence is a
    sarcomere that assembles and transmits force imperfectly and that senses
    mechanical load abnormally - a contractile and mechanosensing deficit rather
    than the hypercontractility of the hypertrophic sarcomeropathies. Human heart
    tissue analyses integrating RNA and protein data underpin the discrimination
    of pathogenic from benign truncations on this basis.
  cell_types:
  - preferred_term: Cardiomyocyte
    term:
      id: CL:0000746
      label: cardiac muscle cell
  biological_processes:
  - preferred_term: Cardiac Myofibril Assembly
    term:
      id: GO:0055003
      label: cardiac myofibril assembly
    modifier: ABNORMAL
  - preferred_term: Muscle Contraction
    term:
      id: GO:0006936
      label: muscle contraction
    modifier: DECREASED
  evidence:
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      we coupled TTN gene sequencing with cardiac phenotyping in 5267 individuals
      across the spectrum of cardiac physiology and integrated these data with RNA
      and protein analyses of human heart tissues
    explanation: >-
      Describes the integrated human tissue RNA/protein analysis on which the
      molecular consequences of TTN truncation in this node rest.
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      A-band are excluded by alternate splicing, or whether the pathogenesis of TTN
      DCM is determined either by critical functional elements located in the
      A-band, or by the length of a truncated TTN protein product.
    explanation: >-
      Records the open question this node deliberately does not over-resolve -
      whether pathogenicity follows from A-band functional elements or from
      truncated-product length. PARTIAL because the mechanism is stated as
      alternatives rather than settled.
  downstream:
  - target: Ventricular Dilation and Systolic Dysfunction
    causal_link_type: DIRECT
    description: >-
      A sarcomere that generates and transmits force poorly dilates and loses
      systolic performance under load.
- name: Ventricular Dilation and Systolic Dysfunction
  biological_scale: TISSUE
  role: central_effector
  conforms_to: "cardiomyopathy_maladaptive_remodeling#Ventricular Remodeling"
  description: >-
    Chronic sarcomeric insufficiency produces the defining structural remodeling of
    dilated cardiomyopathy: progressive left ventricular chamber enlargement with
    wall thinning, myocyte loss, and interstitial fibrosis, accompanied by falling
    ejection fraction. Unlike the hypertrophic sarcomeropathies, the geometric
    response is dilation rather than wall thickening, and neurohormonal activation
    drives the remodeling forward.
  cell_types:
  - preferred_term: Cardiomyocyte
    term:
      id: CL:0000746
      label: cardiac muscle cell
  - preferred_term: Cardiac Fibroblast
    term:
      id: CL:0002548
      label: fibroblast of cardiac tissue
  biological_processes:
  - preferred_term: Heart Contraction
    term:
      id: GO:0060047
      label: heart contraction
    modifier: DECREASED
  - preferred_term: Extracellular Matrix Organization
    term:
      id: GO:0030198
      label: extracellular matrix organization
    modifier: INCREASED
  locations:
  - preferred_term: Left ventricle
    term:
      id: UBERON:0002084
      label: heart left ventricle
  evidence:
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      TTNtv is the most common genetic cause of DCM in ambulant patients in the
      community, identify clinically important manifestations of TTNtv-positive
      DCM, and define the penetrance and outcomes of TTNtv in the general
      population.
    explanation: >-
      Establishes TTNtv as the leading genetic cause of the dilated cardiomyopathy
      phenotype this node represents, in a community-ascertained cohort.
  downstream:
  - target: Heart Failure and Arrhythmic Risk
    causal_link_type: DIRECT
- name: Heart Failure and Arrhythmic Risk
  biological_scale: ORGANISM
  role: consequence
  conforms_to: "cardiomyopathy_maladaptive_remodeling#Structural Cardiac Impairment and Heart Failure"
  description: >-
    The dilated, poorly contracting, fibrotic ventricle produces the clinical
    endpoints of CMD1G: progressive systolic heart failure requiring standard
    heart-failure therapy and, in advanced disease, mechanical support or
    transplantation. TTNtv-positive DCM additionally carries a clinically important
    burden of atrial and ventricular arrhythmia, so rhythm surveillance is part of
    management rather than an incidental concern.
  cell_types:
  - preferred_term: Cardiomyocyte
    term:
      id: CL:0000746
      label: cardiac muscle cell
  biological_processes:
  - preferred_term: Heart Contraction
    term:
      id: GO:0060047
      label: heart contraction
    modifier: ABNORMAL
  evidence:
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      identify clinically important manifestations of TTNtv-positive DCM, and
      define the penetrance and outcomes of TTNtv in the general population.
    explanation: >-
      Anchors the clinically important manifestations and outcomes of TTNtv-positive
      dilated cardiomyopathy that constitute this endpoint node.
phenotypes:
- name: Dilated Cardiomyopathy
  category: Cardiovascular
  description: >-
    Left ventricular dilation with systolic dysfunction, not explained by loading
    conditions or coronary disease, is the defining feature of CMD1G.
  phenotype_term:
    preferred_term: Dilated cardiomyopathy
    term:
      id: HP:0001644
      label: Dilated cardiomyopathy
  frequency: OBLIGATE
  evidence:
  - reference: PMID:22335739
    reference_title: "Truncations of titin causing dilated cardiomyopathy."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      TTN truncating mutations are a common cause of dilated cardiomyopathy,
      occurring in approximately 25% of familial cases of idiopathic dilated
      cardiomyopathy and in 18% of sporadic cases.
    explanation: >-
      Establishes dilated cardiomyopathy as the phenotype caused by TTN truncating
      variants.
- name: Congestive Heart Failure
  category: Cardiovascular
  description: >-
    Progressive systolic dysfunction produces heart failure; dilated cardiomyopathy
    is the commonest precipitating cause of heart transplantation.
  phenotype_term:
    preferred_term: Congestive heart failure
    term:
      id: HP:0001635
      label: Congestive heart failure
  evidence:
  - reference: PMID:21353195
    reference_title: "Genome-wide studies of copy number variation and exome sequencing identify rare variants in BAG3 as a cause of dilated cardiomyopathy."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Dilated cardiomyopathy commonly causes heart failure and is the most frequent
      precipitating cause of heart transplantation.
    explanation: >-
      States the heart-failure and transplantation burden of dilated cardiomyopathy,
      the endpoint this phenotype records. Cited from a DCM gene-discovery paper
      that states the general clinical burden.
- name: Ventricular Arrhythmia
  category: Cardiovascular
  description: >-
    TTNtv-positive dilated cardiomyopathy carries a clinically important burden of
    ventricular arrhythmia on the dilated, fibrotic substrate.
  phenotype_term:
    preferred_term: Ventricular arrhythmia
    term:
      id: HP:0004308
      label: Ventricular arrhythmia
  evidence:
  - reference: PMID:25589632
    reference_title: "Integrated allelic, transcriptional, and phenomic dissection of the cardiac effects of titin truncations in health and disease."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      identify clinically important manifestations of TTNtv-positive DCM, and
      define the penetrance and outcomes of TTNtv in the general population.
    explanation: >-
      The community cohort defining the clinically important manifestations of
      TTNtv-positive DCM, among which arrhythmia is prominent; PARTIAL because the
      quoted sentence names the manifestations collectively rather than arrhythmia
      specifically.
genetic:
- name: TTN
  gene_term:
    preferred_term: TTN
    term:
      id: hgnc:12403
      label: TTN
  relationship_type: CAUSATIVE
  frequency: >-
    The largest single genetic contributor to dilated cardiomyopathy:
    truncating variants account for roughly a quarter of familial and a fifth
    of sporadic idiopathic cases.
  case_fractions:
  - population: Familial idiopathic dilated cardiomyopathy
    case_fraction_percent: 25.0
    notes: >-
      Share of familial idiopathic dilated cardiomyopathy attributable to
      titin truncating variants in the founding study. Pathogenicity is
      position-dependent, so this figure applies to TTNtv as ascertained there
      and not to any truncating variant found incidentally.
    evidence:
    - reference: PMID:22335739
      reference_title: "Truncations of titin causing dilated cardiomyopathy."
      supports: SUPPORT
      evidence_source: HUMAN_CLINICAL
      snippet: >-
        TTN truncating mutations are a common cause of dilated cardiomyopathy,
        occurring in approximately 25% of familial cases of idiopathic dilated
        cardiomyopathy and in 18% of sporadic cases.
      explanation: >-
        Directly states the familial share of cases attributable to TTN
        truncating variants.
  - population: Sporadic idiopathic dilated cardiomyopathy
    case_fraction_percent: 18.0
    notes: >-
      Share of sporadic idiopathic dilated cardiomyopathy attributable to
      titin truncating variants in the same study; recorded separately from
      the familial figure because the two denominators are different
      populations.
    evidence:
    - reference: PMID:22335739
      reference_title: "Truncations of titin causing dilated cardiomyopathy."
      supports: SUPPORT
      evidence_source: HUMAN_CLINICAL
      snippet: >-
        TTN truncating mutations are a common cause of dilated cardiomyopathy,
        occurring in approximately 25% of familial cases of idiopathic dilated
        cardiomyopathy and in 18% of sporadic cases.
      explanation: >-
        Directly states the sporadic share of cases attributable to TTN
        truncating variants.
  evidence:
  - reference: CGGV:assertion_1ec53217-814e-44b3-a7b7-0f18311c20f3-2025-05-30T160000.000Z
    reference_title: "TTN / dilated cardiomyopathy (Definitive)"
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      TTN | HGNC:12403 | dilated cardiomyopathy | MONDO:0005021 | AD |
      Definitive
    explanation: >-
      ClinGen's Gene Curation Expert Panel classifies this gene-disease
      relationship as Definitive, which is the authority for curating the gene
      as CAUSATIVE rather than a candidate.
  - reference: PMID:22335739
    reference_title: "Truncations of titin causing dilated cardiomyopathy."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      the penetrance of TTN truncating mutations was more than 95%
    explanation: >-
      Gene-level penetrance, quoted with the caveat that matters clinically:
      this is the figure among family members over 40 in clinically
      ascertained families, and penetrance among TTNtv carriers ascertained
      from the general population is substantially lower. PARTIAL because the
      number cannot be transferred to an incidentally genotyped individual.
treatments:
- name: Heart Failure Pharmacotherapy
  description: >-
    Standard guideline-directed heart-failure therapy (renin-angiotensin-aldosterone
    system inhibition, beta-blockade, mineralocorticoid receptor antagonism)
    targets the neurohormonal drive of adverse remodeling.
  therapeutic_modality: SMALL_MOLECULE
  treatment_term:
    preferred_term: Pharmacotherapy
    term:
      id: NCIT:C15986
      label: Pharmacotherapy
    therapeutic_agent:
    - preferred_term: renin-angiotensin-aldosterone system inhibitor
      term:
        id: NCIT:C247
        label: ACE Inhibitor
    - preferred_term: beta-blocker
      term:
        id: NCIT:C29576
        label: Beta-Adrenergic Antagonist
    - preferred_term: mineralocorticoid receptor antagonist
      term:
        id: NCIT:C101255
        label: Aldosterone Receptor Antagonist
- name: Implantable Cardioverter-Defibrillator
  description: >-
    Considered for prevention of sudden cardiac death given the arrhythmic burden
    of TTNtv-positive dilated cardiomyopathy.
  therapeutic_modality: DEVICE
  treatment_term:
    preferred_term: implantable cardioverter-defibrillator placement
    term:
      id: NCIT:C80435
      label: Implantable Cardioverter-Defibrillator Placement
- name: Heart Transplantation
  description: >-
    Advanced, refractory disease is an indication for transplantation; dilated
    cardiomyopathy is the most frequent precipitating cause of heart transplant.
  therapeutic_modality: SURGERY
  treatment_term:
    preferred_term: organ transplantation
    term:
      id: NCIT:C15289
      label: Organ Transplantation
references:
- reference: PMID:20301486
  title: Dilated Cardiomyopathy Overview.
  tags:
  - GeneReviews
notes: >-
  CMD1G is curated as the TTN member of the numbered dilated cardiomyopathy series
  and is the highest-value DCM gene entry: TTN truncating variants are the single
  commonest genetic cause of the disease (~25% familial, ~18% sporadic), and TTN
  carries ClinGen Definitive gene-disease validity for DCM. It conforms to the
  shared cardiomyopathy_maladaptive_remodeling module.

  Positional caveat preserved on the pathograph, deliberately. TTNtv occur in the
  general population, so a truncating variant is not by itself a diagnosis;
  pathogenicity tracks with A-band location. The entry also records the unresolved
  question (PMID:25589632) of whether pathogenicity follows from critical
  functional elements in the A-band or from the length of the truncated product,
  rather than asserting a single settled mechanism. Penetrance is curated with the
  ascertainment caveat: >95% in clinically ascertained families over 40, but
  substantially lower among population-ascertained carriers.

  Note the contrast with Hypertrophic Cardiomyopathy 9 (also TTN): TTN truncating
  variants are established in dilated, not hypertrophic, cardiomyopathy, and the
  TTN-HCM association rests on rare missense reports - a caveat already recorded on
  that entry and in the Familial Hypertrophic Cardiomyopathy grouping.

  GeneReviews scope. The GeneReviews resource applicable to this entry is the
  disease-level "Dilated Cardiomyopathy Overview" (PMID:20301486), tagged
  accordingly in `references`. Its indexed PubMed record is content_type
  abstract_only and carries only the chapter's purpose statement, not the
  Clinical Characteristics, Management, or Genetic Counseling sections, so
  section-by-section GeneReviews mining is not possible from the cache and no
  snippet is quoted from it. The clinical-characteristics baseline for this
  entry is therefore built from the primary TTN cohort and pedigree literature
  cited throughout, principally PMID:22335739, PMID:21353195, PMID:25589632.
📚

References & Deep Research

References

1
Dilated Cardiomyopathy Overview.
No top-level findings curated for this source.