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Target of EGR1 protein 1 (TOE1) is a nuclear RNA exonuclease and member of the deadenylase family, primarily functioning in the 3'-end maturation of small nuclear RNAs (snRNAs)[2][4]. It possesses poly(A)-specific ribonuclease activity, regulating RNA turnover and metabolism. TOE1 modulates cell cycle progression, inhibits cell proliferation, and regulates neural progenitor proliferation and differentiation; loss of function is linked to pontocerebellar hypoplasia type 7, a severe infant neurodegenerative syndrome[2][4]. TOE1 also localizes to Cajal bodies and is implicated in RNA splicing. Notably, TOE1 can inhibit HIV-1 replication by binding to viral RNA and regulating transcriptional responses, suggesting a role in immune antiviral defense[1]. TOE1 is often referred to as "Target of EGR1 protein 1" (abbreviation: TOE1), and is not commonly known as a druggable therapeutic target, but it holds biological significance in cancer, neurodegeneration, and antiviral immunity[1][2][4]. The protein lacks currently known direct drug modulators, standardized biomarkers, or established mechanisms of action for therapeutics targeting TOE1[1][2][4].
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