Ribonucleic acid and Precursor messenger RNA (RNA and pre-mRNA)

RNA (ribonucleic acid) is a nucleic acid polymer composed of ribonucleotides that serves multiple roles in the cell, including the transfer of genetic information, catalytic activity, and regulation of gene expression. Precursor messenger RNA (pre-mRNA) is a primary transcript synthesized by RNA polymerase II in eukaryotes; it undergoes extensive processing—5' capping, splicing to remove introns, and 3' polyadenylation—to generate mature messenger RNA (mRNA), which serves as the template for protein synthesis. Both RNA and pre-mRNA are essential intermediates in gene expression and are dynamic participants in the regulation of splicing and other post-transcriptional modifications. Although not usually considered specific drug targets in the sense of a receptor or enzyme, disease-causing or disease-associated RNAs, including pre-mRNAs, can be modulated by antisense oligonucleotides, small molecules, and RNA interference-based therapeutics. The versatility, structural complexity, and fundamental role of RNA in gene regulation and cell biology have made RNA-based therapies and diagnostics an expanding area of biomedical research.

Other names

pre-mRNA: primary transcriptprecursor mRNAhnRNA (heterogeneous nuclear RNA)RNA: ribonucleic acidincludes mRNA, tRNA, rRNA, snRNA, and other non-coding RNAs

Modulation of RNA structure to alter splicing or stability Degradation by RNase H (antisense) Blockage of translation (siRNA, antisense) Alteration of splicing patterns (splice modulators)