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APOBEC1 complementation factor (A1CF) is an essential RNA-binding protein that serves as a core component of the apolipoprotein B (apoB) mRNA editing complex, mediating site-specific cytidine-to-uridine (C-to-U) RNA editing in cooperation with APOBEC1[1][2][5][6]. A1CF contains multiple RNA recognition motifs (RRMs) and directs APOBEC1 to specific target cytidines, playing a critical role in post-transcriptional gene regulation including mRNA editing, stability, and nucleocytoplasmic transport[1][3][6]. A1CF is involved in processes such as embryonic development, kidney physiology, and liver regeneration; dysregulation or overexpression of A1CF is associated with metabolic disease, fibrosis, and several cancers, notably hepatocellular carcinoma, where it drives pathogenic pathways of lipogenesis and proliferation[2][4][7]. A1CF functions as a molecular adaptor in RNA-protein complexes and is ubiquitously expressed in many human tissues[1][2][3]. No drugs are known to target this protein directly, but its activity and expression are regulated by hormones and cellular signaling pathways[2].
No drugs currently target A1CF directly; mechanisms linked to the modulation of RNA editing and mRNA stability by protein-protein or protein-RNA binding
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