U1 spliceosomal RNA (U1 snRNA)

U1 spliceosomal RNA is a small nuclear RNA (snRNA) of 164 nucleotides in humans, serving as a critical part of the U1 small nuclear ribonucleoprotein (snRNP) within the spliceosome, the multi-megadalton complex responsible for pre-mRNA splicing in eukaryotic cells. U1 snRNA recognizes and base-pairs with the 5′ splice site of introns in pre-mRNA, thereby initiating spliceosome assembly. The U1 snRNP further consists of the U1 RNA, core Sm proteins, and U1-specific proteins (U1-70K, U1-A, and U1-C), which collectively mediate splice site recognition and facilitate subsequent splicing reactions. Structural studies have elucidated stem-loop motifs and complex RNA-protein interactions that are evolutionarily conserved. Recent research has revealed additional roles for U1 snRNP in regulation of alternative polyadenylation and transcript processing. Mutations or misregulation of spliceosomal RNA components can lead to aberrant splicing and disease states, including various cancers and neurodegenerative conditions. While not directly used as a biomarker, aberrant splicing activity involving U1 snRNA may serve as a molecular indicator of splicing defects or disease.

Other names

U1 spliceosomal RNAU1 snRNAU1 small nuclear RNAsnRNA U1U1 snRNP RNA component

N/A. No drugs are known to target this RNA directly; some experimental oligonucleotides (antisense morpholinos) have been used to modulate its activity in research settings, but these are not approved therapies.