Human nucleic acids (DNA/RNA)

Human nucleic acids, comprising deoxyribonucleic acid (DNA) and ribonucleic acid (RNA), serve as the fundamental repositories and transmitters of genetic information within the cell [National Human Genome Research Institute (NHGRI)]. DNA primarily functions in the long-term storage of genetic blueprints, while various forms of RNA, including mRNA, tRNA, and rRNA, facilitate the translation of these blueprints into functional proteins [Nature Education]. In the context of pharmacology, nucleic acids are targeted by a wide array of therapeutic agents; for instance, traditional chemotherapeutics like cisplatin cross-link DNA to induce apoptosis in rapidly dividing cancer cells [PubChem]. Modern therapeutic strategies have expanded to include antisense oligonucleotides and small interfering RNAs (siRNAs) that specifically modulate RNA stability or translation to treat genetic and metabolic disorders [Nature Reviews Drug Discovery]. Despite their central role in therapy, targeting nucleic acids poses significant challenges, including the risk of secondary malignancies due to genomic instability and the difficulty of achieving sequence-specific delivery [American Cancer Society].

Other names

Deoxyribonucleic acidRibonucleic acidGenomic DNACellular RNAGenetic materialPolynucleotide

Mechanisms include DNA alkylation, intercalation, strand cross-linking, antimetabolite incorporation, antisense inhibition, and RNA interference [Nature Reviews Drug Discovery, PubChem].