What is the goal of gene therapy?

The primary goal of gene therapy is to replace defective genes with functional ones. This approach aims to address genetic disorders by correcting underlying genetic deficiencies caused by mutations. By introducing a healthy copy of a gene into the patient's cells, gene therapy seeks to restore normal function and alleviate the symptoms of genetic diseases. It can lead to the production of functional proteins that are essential for normal cellular processes, thereby improving overall health outcomes for patients with inherited genetic conditions.

While the other options mention important biological processes, they do not encapsulate the fundamental aim of gene therapy. Enhancing protein synthesis might be a secondary effect of successful gene therapy, but it is not the therapy’s primary intention. Modifying RNA functionality can play a role in other therapeutic strategies but does not directly address the replacement of defective genes. Inhibiting viral replication is critical in the context of antiviral therapies, rather than gene therapy specifically, which focuses on genetic defects in cells. Thus, replacing defective genes with functional counterparts is the definitive goal of gene therapy.