U2 small nuclear ribonucleoprotein (U2 snRNP)

The **U2 small nuclear ribonucleoprotein** is a critical multi-protein–RNA complex within the major spliceosome, which is responsible for pre-mRNA splicing in all eukaryotes[2][3][7][9]. U2 snRNP contains the U2 small nuclear RNA (snRNA), a set of Sm proteins (U2A′, U2B″), and protein subcomplexes SF3A and SF3B[2][4][5]. Its primary function is to recognize the branchpoint sequence of introns via base pairing, enabling selection of the branchpoint adenosine essential for the first step of splicing[2][7]. U2 snRNP undergoes dynamic conformational and compositional changes as the spliceosome assembles and becomes catalytically active[3][8]. The SF3B component, particularly SF3B1, is a major target for cancer-associated mutations and for splicing modulator drugs, highlighting both its fundamental biological and clinical significance[1][3][7]. The structure and function of U2 snRNP are highly conserved, and defects in its function or regulation are implicated in various cancers and possibly other diseases[1][3][8]. U2 snRNP is not directly considered a "therapeutic target" in the classic sense (like a receptor or enzyme), but modulators targeting it are investigated for cancer treatment, and its components are important molecular markers in disease research[1][3][7][8].

Other names

U2 snRNPU2 spliceosomal small nuclear ribonucleoproteinU2 spliceosomal RNA (when referring to the RNA component alone)U2 spliceosomal particle

Modulation of spliceosome assembly and function (by small-molecule inhibitors) Disruption of branchpoint recognition or SF3B1 activity, altering pre-mRNA splicing specificity or efficiency