How is gene therapy defined?

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Gene therapy is defined as a method that alters or replaces defective genes. This approach is designed to address genetic disorders caused by mutations or defective genes, thereby providing a potential cure or mitigation for these conditions. The focus of gene therapy is on correcting the underlying genetic issues that lead to disease, which often involves inserting healthy copies of a gene into a patient's cells, repairing or replacing faulty genes, or directly modifying the expression of genes to restore normal function.

This definition emphasizes the therapeutic intent and application of gene therapy in treating diseases, particularly those that have a genetic basis. By targeting the genetic root of a disorder, gene therapy has the potential to offer long-term solutions rather than merely treating symptoms. This sets it apart from techniques that modify gene functions through enzymatic means, cloning for research purposes, or creating synthetic genes, which do not directly aim to correct inherited genetic defects in patients.

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