Gene replacement or correction (None established; sometimes referred to as "gene therapy" or "genome editing" in the literature, but no standard abbreviation for this specific process.)

Gene replacement/correction refers broadly to therapeutic strategies that aim either to introduce functional copies of defective genes into patient cells ("gene addition") or directly correct pathogenic DNA changes ("genome editing"). These approaches can be implemented using viral vectors engineered to deliver healthy gene sequences or through precise genome-editing technologies like CRISPR-Cas9 that modify endogenous DNA at targeted locations. The goal is restoration—or compensation—of normal cellular function lost due to inherited mutations. While highly promising for monogenic disorders and some acquired conditions like cancer, these interventions are complex procedures rather than discrete molecular targets themselves and require careful patient selection based on underlying genetics along with ongoing safety monitoring due to risks such as immune reactions and off-target genomic alterations.

Other names

Gene therapyGenome editingGenetic correctionGene addition/replacement

Mechanisms depend on the technology used: - Replacement: Introducing functional copies of faulty genes into cells using viral/nonviral vectors. - Correction/editing: Directly repairing DNA sequence errors via genome editing tools such as CRISPR-Cas9, TALENs, zinc finger nucleases. - Knockout/inactivation: Disabling malfunctioning genes causing disease. These approaches aim to restore normal protein production/function within affected cells/tissues