Pre-mRNA processing factor 4 (PRPF4)

Pre-mRNA processing factor 4 (PRPF4) is a conserved spliceosomal protein, essential for the formation and activity of the U4/U6-U5 tri-snRNP complex within the spliceosome, which catalyzes intron removal from pre-messenger RNA transcripts. PRPF4 forms stable interactions with peptidyl-prolyl isomerase H (PPIH) via bipartite binding involving intrinsically disordered regions, is central to spliceosome assembly and regulation, and supports alternative splicing. Mutations in PRPF4 destabilize the spliceosome and impair splicing, leading to genetic diseases such as autosomal dominant retinitis pigmentosa; PRPF4 also plays roles in chemoresistance and cell cycle regulation. While essential for normal cell biology, PRPF4 has not yet been systematically targeted by therapeutics, in part due to potential toxicity risks from globally disrupting splicing.

Other names

U4/U6 small nuclear ribonucleoprotein Prp4PRP4hPrp4Prp4pHPRP4HPRP4PSNRNP60PRP4 homologU4/U6 snRNP 60 kDa proteinWD splicing factor Prp4PRP4/STK/WD splicing factorPRP4 pre-mRNA processing factor 4 homologpre-mRNA splicing tri-snRNP complex factor PRPF4RP70

Not applicable—currently, there are no established drugs that directly target PRPF4. For potential approaches: gene therapy, RNA/spliceosome modulators, or small molecules that might impact the spliceosome or correct splicing defects are hypothetical.