Target intelligence / Profile preview

Fanconi anemia group C protein (FANCC)

Target
FANCC
Molecular classification
DNA repair protein, Fanconi anemia core complex subunit, Other (multisubunit DNA damage response protein complex member)
01

Overview

Fanconi anemia group C protein (FANCC) is a key subunit of the FA core complex involved in DNA damage response, especially the repair of interstrand cross-links that block DNA replication. FANCC regulates the recruitment and activation of other FA proteins such as FANCD2 and is essential for the stability and function of the multisubunit repair complex. Deficiency or mutation leads to Fanconi anemia, a syndrome characterized by bone marrow failure, congenital abnormalities, and cancer predisposition. FANCC also has roles in cytokine signaling (STAT1 pathway), linking DNA repair to immune response and apoptosis suppression. FANCC interacts with other FA proteins (FANCA, FANCG) and is found in both cytoplasm and nucleus as part of the core complex. There are no approved drugs that specifically target FANCC, but its functional status may inform chemotherapy choice and patient management.

Other names
Fanconi anemia group C proteinFANCCFACFACCProtein FACCFA3LOC105376156
02

Mechanism of action

No drug directly targeting FANCC is clinically approved. In research, disruption of the FA core complex (e.g., small molecules interfering with protein-protein interactions) has been proposed to sensitize cancer cells to DNA cross-linking agents. Cellular response to DNA cross-linking agents relies on FANCC-dependent repair; thus, loss of FANCC function leads to increased sensitivity.

03

Biological functions

DNA damage repair (specifically interstrand cross-link repair)Activation of STAT1 signalingApoptosis suppression (modulation of cytokine-induced apoptosis)Cell cycle checkpoint control (as part of the FA pathway)
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Disease associations

Cancer predisposition (especially hematologic malignancies)Bone marrow failure syndromes (Fanconi anemia)Others related to genomic instability
05

Safety considerations

Patients and cells deficient in FANCC are highly sensitive to DNA cross-linking chemotherapyIncreased risk of bone marrow failure, severe cytopenias, and cancerOff-target inhibition or deficiency can result in genomic instability, pancytopenia, and predisposition to malignancyTargeting FANCC for therapy is challenging due to its essential role in normal DNA repair and hematopoiesis
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Interacting drugs

DNA cross-linking agents (e.g., mitomycin C, cyclophosphamide; these drugs reveal cellular hypersensitivity in FANCC-deficient cells but are not direct protein-drug binders)
07

Biomarkers

Germline or somatic mutation in FANCC (used to diagnose Fanconi anemia, stratify risk, and explain hypersensitivity to DNA-damaging agents)Cellular hypersensitivity to mitomycin C is a functional diagnostic test for FA, including FA-C subgroup

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