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KH-type splicing regulatory protein (KHSRP) is a multifunctional RNA-binding protein that modulates gene expression at the levels of pre-mRNA splicing, mRNA decay, and microRNA maturation. It preferentially binds single-stranded nucleic acids via four KH-domains, promoting rapid degradation of labile transcripts containing AU-rich elements and facilitating the maturation of select microRNAs. As a regulator of critical cellular processes—including cell proliferation, differentiation, immune response, and metabolism—KHSRP is implicated in diverse diseases such as cancer, neuromuscular disorders, inflammation, obesity, and diabetes. KHSRP's complex regulatory roles make it a significant potential therapeutic target, although direct drugs have yet to be established[1][2][3][4][5].
Not applicable (no direct drugs known; hypothetical strategies include modulation of RNA-binding, inhibition of mRNA decay function, or alteration of miRNA maturation[1])
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