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Pre-mRNA-splicing factor ATP-dependent RNA helicase PRP16 (DHX38) is an ATPase and RNA helicase belonging to the DEAD/H box family, acting as a key component of the spliceosome and catalyzing the second step of pre-mRNA splicing essential for exon ligation. DHX38 is involved in maintaining splicing fidelity, alternative splicing of mitosis-related genes, and genomic stability by preventing R-loop accumulation and DNA replication stress. Mutations in DHX38 are associated with autosomal recessive retinitis pigmentosa and the protein is required for cell survival and proliferation in various tissues, including the retina and certain cancers[1][3][4][5]. Key evidence links DHX38 with both developmental processes in the retina and tumor biology, highlighting its importance in cell homeostasis, gene regulation via splicing, and as a potential target in relevant diseases[1][3][5]. Direct targeting by drugs is not documented, but its centrality to splicing suggests any therapeutic modulation would require careful management due to possible effects on genomic stability and cell viability.
Not established for direct drugs; DHX38's function as RNA helicase and spliceosomal enzyme is targeted in genetic models or experimental contexts by knockdown, mutation, or possibly splicing modulator compounds
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