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Pre-mRNA-splicing factor SYF1 (XAB2) is a conserved, multifunctional nuclear protein of approximately 100 kDa, featuring 15 tetratricopeptide repeat (TPR) motifs that mediate protein–protein interactions[1][2]. XAB2 participates in several essential processes: it is a component of the Prp19/XAB2 complex required for pre-mRNA splicing; it plays a critical, specific role in transcription-coupled nucleotide excision repair (TC-NER), especially for RNA polymerase II-transcribed genes[1][2][3]. It also regulates transcription of certain genes (e.g., CENPE, contributing to cell cycle progression), mRNA export, and homologous recombination repair[1][2][3]. Loss of XAB2 results in impaired splicing, defective TC-NER, genome instability, mitotic arrest, and cell death[1][2]. The protein is implicated in cancer biology due to its essential roles in DNA repair and genome stability, and is crucial for early embryogenesis[1][2][3]. No clinically approved drugs or specific small molecules are known to target XAB2 directly, and no validated biomarkers or mechanisms of drug action specific to XAB2 have been described in the literature[1][2][3]. Note: XAB2 is not a classical therapeutic target (e.g. receptor, enzyme, or transporter) but rather a multifunctional splicing and DNA repair factor necessary for cell viability and genome integrity[1][2][3].
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