RNA, U5A small nuclear 1 (RNU5A-1)

RNA, U5A small nuclear 1 (RNU5A-1) encodes the U5A variant of small nuclear RNA (snRNA), a non-coding, uridine-rich RNA essential for the function of the major spliceosome in eukaryotic cells[1][4][7]. U5 snRNA forms part of the U4/U5/U6 tri-snRNP complex, which mediates the removal of introns from pre-messenger RNA through the splicing reaction[1][7]. The U5 snRNA specifically plays a critical role in aligning exons by binding to both the 5' and 3' splice sites, ensuring precise ligation during catalysis[5]. Variants in RNU5A-1 have been associated with neurodevelopmental disorders, as pathogenic de novo mutations are observed in patients with clinical features consistent with splicing defects, making it a strong candidate gene but not an established therapeutic target[5]. U5 snRNA, like other snRNAs, functions as part of small nuclear ribonucleoproteins (snRNPs) and does not code for proteins nor act as a receptor, enzyme, or classical drug target[1][4]. There are currently no drugs that directly target RNU5A-1, and no known biomarkers or safety concerns linked to direct manipulation of this molecule.

Other names

U5AU5B1RNU5RNU5ARNU5CU5CRNA, U5C small nuclearRNU5A-1