Cellular Nucleic Acids (DNA and RNA) (DNA/RNA)

Cellular DNA and RNA, collectively known as nucleic acids, serve as the fundamental blueprints and functional templates for life. DNA stores the hereditary information required for the development and functioning of all living organisms, while RNA plays critical roles in translating that information into proteins and regulating gene expression (Source: NIH). In the context of pharmacology, these molecules are primary targets for a wide range of therapeutic agents. Traditional chemotherapies often target DNA through alkylation or intercalation to induce cell death in rapidly dividing cancer cells, particularly in oncology (Source: PubMed). Modern therapeutics, such as antisense oligonucleotides and siRNA, target specific RNA sequences to modulate protein production for treating genetic and rare diseases (Source: Nature Reviews Drug Discovery). Despite their therapeutic utility, targeting nucleic acids poses significant safety challenges, including the risk of off-target genetic damage and secondary malignancies due to their ubiquitous presence and essential nature.

Other names

Deoxyribonucleic acidRibonucleic acidGenetic materialPolynucleotides

Drugs targeting nucleic acids act through various mechanisms including DNA alkylation (cross-linking strands), intercalation (inserting between base pairs), inhibition of topoisomerases, antimetabolite incorporation (mimicking nucleotides), and RNA-targeted approaches like antisense oligonucleotides (ASOs) or RNA interference (RNAi) to modulate translation (Source: NIH, PubMed).