Is gene therapy being used to cure diseases? What is its promise for the future of medicine?
Gene therapy is very young and experimental. Many factors have prevented researchers from developing successful gene therapy techniques. The first hurdle is the gene delivery tool. How is a new gene inserted into the body? This is done via vehicles called vectors (gene carriers), which deliver therapeutic genes to the patients’ cells. Currently, the most common vectors are viruses. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to take advantage of the virus’s biology and manipulate its genome to remove the disease-causing genes and insert therapeutic genes. Viruses, while effective, introduce other problems to the body –toxicity, immune and inflammatory responses, and gene control and targeting issues. Some alternatives to viruses that have been considered are complexes of DNA with lipids and proteins. Researchers are also experimenting with introducing a 47th (artificial human) chromosome to the bod
