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KH domain-containing, RNA-binding, signal transduction-associated protein 1 (Sam68) is a multifunctional protein first identified as the 68-kDa substrate for Src family tyrosine kinases during mitosis[4][1][7]. It contains a GSG domain (also called the STAR domain), central KH RNA-binding motif, and several proline- and tyrosine-rich regions that mediate interactions with SH2/SH3 domain-containing signaling proteins[4][5][8]. Sam68 regulates pre-mRNA alternative splicing, RNA metabolism, cell cycle progression, and apoptosis[4][5][2]. It acts as a signaling adaptor and links extracellular signals to RNA processing events. Sam68 is involved in cell proliferation, cytoskeletal dynamics, and viral replication (notably, it is essential for the function of HIV-1 Rev protein)[7]. Overexpression or aberrant activity of Sam68 has been linked to cancer progression in multiple tissue types, making it a potential biomarker and therapeutic target for oncology, though direct inhibitors are not clinically available[4][5].
Not applicable for direct drugs, but Sam68 acts as a multifunctional adapter and RNA-binding protein, modulating alternative splicing, RNA localization, and signal relay.
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