Deoxyribonucleic acid and proteins

Deoxyribonucleic acid (DNA) and proteins constitute the two primary classes of biological macromolecules that serve as the foundation for nearly all therapeutic interventions. DNA serves as the primary repository of genetic information, and its targeting is a cornerstone of traditional chemotherapy, where agents like alkylators and intercalators disrupt cellular replication to treat various cancers (National Cancer Institute, 2023). Proteins, which include enzymes, receptors, ion channels, and transporters, are the functional workhorses of the cell and represent the most diverse class of drug targets (Santos et al., 2017). Therapeutic strategies involving proteins range from small-molecule inhibition of enzymatic activity to monoclonal antibodies that block cell-surface receptors or neutralize circulating ligands. Because this designation combines two distinct and massive classes of biological molecules, it is generally classified as a broad category rather than a specific, singular target for drug development. Consequently, identifying a specific gene product or protein isoform is necessary for precise pharmacological characterization and clinical application.

Other names

Nucleic acids and polypeptidesBiomacromoleculesGenetic material and cellular machinery

Drugs targeting DNA typically involve intercalation, alkylation, or strand breakage to inhibit replication and transcription (StatPearls, 2024). Drugs targeting proteins act via competitive or non-competitive inhibition, agonism, antagonism, or by modulating protein-protein interactions and degradation pathways (Santos et al., 2017).