Nucleic acids and associated proteins

Nucleic acids and associated proteins represent the collective machinery of the central dogma of molecular biology, encompassing the storage, transmission, and execution of genetic information (Nature Reviews Drug Discovery, 2018). DNA serves as the stable template for heredity, while various forms of RNA, including mRNA and non-coding RNA, facilitate protein synthesis and regulatory functions (NIH, 2023). Associated proteins, such as histones for structural packaging and enzymes like polymerases and topoisomerases for replication and transcription, are critical for maintaining genomic integrity and accessibility (UniProt, 2024). In clinical contexts, this broad system is the focus of numerous therapeutic strategies, ranging from traditional cytotoxic chemotherapies that damage DNA to modern precision medicines like PARP inhibitors and antisense oligonucleotides (PubMed, 2022). Dysregulation within this system is a hallmark of cancer, viral infections, and hereditary genetic diseases, making these molecules some of the most historically significant and diverse targets in pharmacology (StatPearls, 2023). However, because these components are ubiquitous and essential for normal cell survival, therapeutic interventions often carry risks of systemic toxicity and long-term genotoxicity (FDA, 2021).

Other names

Genetic materialChromatin and ribonucleoprotein complexesGenome and transcriptomeNucleic acids and associated proteins

Drugs targeting this complex system act through various mechanisms including DNA cross-linking, intercalation, inhibition of topoisomerases or polymerases, antisense-mediated RNA degradation, and modulation of epigenetic marks.