Final answer:
Gene therapy is a genetic engineering technique to treat genetic diseases by introducing a healthy gene into the cells of individuals with a mutated gene. It uses viral vectors for delivery and focuses on single-gene disorders. Though promising, it is still experimental and improving in terms of safety and effectiveness.
Step-by-step explanation:
What is Gene Therapy?
Gene therapy is a revolutionary genetic engineering technique aimed at treating genetic diseases. It involves the process of transferring a healthy, non-mutated gene into the cells of an individual with a mutated gene responsible for a disease. This healthy gene can be delivered through a viral vector, such as an adenovirus, which has been modified to carry the gene instead of viral DNA. Gene therapy strategies vary, but they generally fall into two categories: those that introduce a gene at a random location within the genome, and the more advanced forms that correct the gene at its original location. This field is particularly focused on single-gene disorders like cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia.
The goal of gene therapy is to replace or repair the defective gene so that the body can start to produce the functional protein on its own, potentially curing the genetic disorder. While this technology has shown promise and some success, it is still largely experimental and undergoing development to improve its safety and effectiveness.