What is gene therapy?

Gene therapy is defined as a process of modifying genes to treat or prevent diseases. This technique involves altering the genetic material within a person's cells to correct or replace dysfunctional genes responsible for disease development. By modifying genes, the underlying causes of genetic disorders can be addressed directly, leading to potential long-term solutions for conditions that currently lack effective treatments.

The focus of gene therapy is on therapeutic applications, where the intention is to ameliorate or cure conditions such as genetic disorders, some types of cancer, and viral infections by using techniques that either insert, alter, or remove genes within an individual's cells. This process can involve adding a healthy copy of a gene to compensate for a non-functional gene, repairing a defective gene, or even disrupting the activity of a gene that is harmful.

In contrast, methods geared toward gene identification, cloning, or genetic testing operate under different principles and objectives. Gene identification is focused on discovering and characterizing genes, cloning refers to creating copies of a gene, and genetic testing is conducted to analyze DNA for specific genes or mutations to assess health risks, rather than modifying the genes themselves.