Therapeutic gene

A therapeutic gene is a sequence of nucleic acid (DNA or RNA) introduced into a patient's cells to treat, cure, or prevent disease by altering genetic expression or cellular function (Wikipedia, 2024). This modality serves as the core component of gene therapy, where the gene acts as the therapeutic agent to replace a defective or missing gene, silence a pathogenic gene, or provide a new biological function (NIH, 2023). These genes are delivered to target tissues using various vectors, including viral systems like adeno-associated virus (AAV) or lentivirus, and non-viral systems such as lipid nanoparticles (PMC, 2023). While offering the potential for long-term or even curative outcomes for monogenic disorders (e.g., spinal muscular atrophy, hemophilia) and certain cancers, the use of therapeutic genes involves significant challenges (PubMed, 2000). These include the risk of host immune responses to the vector, potential insertional mutagenesis leading to oncogenesis, and the difficulty of achieving stable, tissue-specific expression (PubMed, 2023). In the context of drug discovery and biotechnology, the 'therapeutic gene' is classified as a therapeutic modality or payload rather than a conventional biological target molecule like a receptor or enzyme (FDA, 2025).

Other names

TransgeneTherapeutic DNATherapeutic RNAGene constructTherapeutic nucleic acid

Modification of the host cellular genetic profile via gene addition, replacement, or silencing to restore physiological function or counteract pathology.