Endogenous ribonucleic acid (RNA) (RNA)

Endogenous ribonucleic acid (RNA) refers to the complete set of RNA molecules naturally produced by a cell's transcriptional machinery, including coding sequences like messenger RNA (mRNA) and non-coding varieties such as microRNA (miRNA) and long non-coding RNA (lncRNA) (Source: Nature Reviews Genetics, 2014). These molecules are fundamental to cellular life, acting as the intermediary template for protein synthesis and serving as sophisticated regulators of gene expression and architectural scaffolds within the nucleus and cytoplasm (Source: Molecular Biology of the Cell, 6th Ed). Pathological alterations in endogenous RNA—such as point mutations, aberrant splicing, or over-expression—underlie a vast array of human diseases, including genetic disorders, cancers, and neurodegenerative conditions (Source: Nature Reviews Drug Discovery, 2020). As a therapeutic target, endogenous RNA offers a way to intervene in disease processes that are "undruggable" at the protein level. Modern pharmacology utilizes antisense oligonucleotides (ASOs), siRNA, and RNA-binding small molecules to selectively degrade toxic transcripts, restore functional protein production through splicing correction, or silence overactive genes, thereby providing highly specific precision medicine approaches (Source: Journal of Medicinal Chemistry, 2021).

Other names

Cellular RNAHost RNATranscriptomeEndogenous transcript

Antisense oligonucleotides (ASOs) bind to target RNA to induce RNase H-mediated degradation or modulate splicing; small interfering RNAs (siRNAs) utilize the RISC complex for targeted mRNA cleavage; small molecules can bind to RNA secondary structures to inhibit translation or modify splicing (Source: Nature Reviews Drug Discovery, 2020).