chromosome 9 open reading frame 72

E561105

Chromosome 9 open reading frame 72 (C9orf72) is a human gene best known for its hexanucleotide repeat expansions that are a major genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

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Observed surface forms (1)

Surface form Occurrences
C9orf72 0

Statements (48)

Predicate Object
instanceOf disease-associated gene
human gene
associatedWithBiologicalProcess autophagy
immune regulation
lysosomal homeostasis
vesicle trafficking
associatedWithClinicalFeature behavioral changes
early-onset dementia
motor neuron degeneration
parkinsonism in some carriers
associatedWithDisease ALS-FTD spectrum disorders
amyotrophic lateral sclerosis
frontotemporal dementia
associatedWithPathology RNA foci
TDP-43 proteinopathy
dipeptide repeat protein aggregates
associatedWithPhenotype ALS with cognitive impairment
behavioral variant frontotemporal dementia
familial ALS
familial frontotemporal dementia
sporadic ALS
encodes C9orf72 protein NERFINISHED
expansionLocation non-coding region
promoter or first intron region
expressedInTissue central nervous system
frontal cortex
motor neurons
fullName chromosome 9 open reading frame 72 NERFINISHED
geneticTestingUse diagnosis of familial ALS
diagnosis of familial frontotemporal dementia
inheritancePattern autosomal dominant
locatedOnChromosome chromosome 9
molecularConsequence haploinsufficiency
toxic RNA gain-of-function
toxic dipeptide repeat protein gain-of-function
mutationTypeInDisease hexanucleotide repeat expansion
normalAlleleFeature short G4C2 repeat length
pathogenicVariantType large hexanucleotide repeat expansion
proteinComplexAssociation SMCR8-C9orf72-WDR41 complex NERFINISHED
proteinFunction endosomal-lysosomal pathway regulation
regulation of autophagy
regulation of membrane trafficking
repeatExpansionEffect aberrant RNA foci formation
production of dipeptide repeat proteins via RAN translation
reduced C9orf72 transcript levels
repeatMotif G4C2
GGGGCC
symbol C9orf72 NERFINISHED

Referenced by (1)

Full triples — surface form annotated when it differs from this entity's canonical label.

C9orf72 fullName chromosome 9 open reading frame 72