Gene

A gene is the fundamental physical and functional unit of heredity, consisting of a specific sequence of nucleotides in DNA or RNA that provides the instructions for synthesizing a functional product, typically a protein or a non-coding RNA molecule (Wikipedia, 2024). In the context of human health, genes serve as the blueprint for all biological processes, and their dysfunction—through mutations, deletions, or aberrant expression—is the underlying cause of numerous conditions, including cancer and inherited genetic disorders (NIH, 2024). While specific genes are the focus of advanced therapeutic interventions such as gene therapy, CRISPR-based gene editing, and antisense oligonucleotides, the term "Gene" itself represents a broad biological category rather than a discrete, individual therapeutic target (NIH, 2023). In pharmacological research, targets are typically defined at the level of specific gene products, such as receptors, enzymes, or transporters, or specific mutated sequences associated with a particular disease (UniProt, 2022). Consequently, "Gene" as a standalone term is considered too generic for precise target annotation in a clinical or drug discovery context.

Other names

Hereditary unitCistronGenetic locusDNA segment

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