Gene therapy is a medical approach that aims to treat genetic disorders by modifying or replacing defective genes with healthy ones. The science behind gene therapy involves the use of vectors, which are vehicles that can deliver therapeutic genes to target cells in the body. The most commonly used vectors for gene therapy are viruses, which have the ability to infect cells and transfer their genetic material into the cell's DNA.
In gene therapy, the viral vector is modified to carry the therapeutic gene instead of its own genes. Once the vector carrying the therapeutic gene enters the target cells, the gene is integrated into the host cell's DNA and begins to produce the functional protein that was missing or defective in the patient's cells.
There are different types of gene therapy, including somatic gene therapy, which targets cells that are not involved in the production of gametes (sperm and egg cells), and germline gene therapy, which targets the DNA of gametes to prevent the transmission of genetic diseases to future generations.
Gene therapy has the potential to cure or alleviate a wide range of genetic diseases, including inherited disorders, cancer, and viral infections. However, it is a relatively new and complex field, and its long-term safety and efficacy are still being studied.