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Target intelligence / Profile preview
Chromosome 9 open reading frame 72 (C9orf72)
- Target
- C9orf72
- Molecular classification
- Other
01
Overview
C9orf72 is a protein involved in several cellular processes including RNA metabolism, vesicle trafficking, autophagy, and immune modulation. A hexanucleotide repeat expansion within the C9orf72 gene is a major genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The expansion leads to both loss-of-function and gain-of-toxicity mechanisms, disrupting cellular homeostasis and contributing to neurodegeneration.
- Other names
- C9orf72-SMCR8 complex subunit
02
Biological functions
Regulation of RNA metabolismVesicle trafficking and lysosome homeostasisAutophagy regulationImmune modulation
03
Disease associations
Amyotrophic Lateral SclerosisFrontotemporal Dementia
04
Safety considerations
Potential for off-target effects when developing therapies that target the GGGGCC repeat expansion.Complexity of addressing both loss-of-function and gain-of-toxicity mechanisms in therapeutic development.
05
Biomarkers
GGGGCC repeat expansion in C9orf72 gene
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