The Pros And Cons Of Gene Therapy

Gene therapy is an experimental treatment, which is used to replace mutated, improperly functioning genes with regular DNA. There are several gene therapy methods, most of which involve using a vector to transport the corrected gene into targeted cells. Retroviruses, adenoviruses, adeno associated viruses and liposomes are commonly used as vectors. Naked DNA (DNA without a vector) has also been used in gene therapy. Recently, gene therapy has been used as a treatment for deadly genetic conditions such as cystic fibrosis. Even though gene therapy has a promising future, it has major disadvantages; including vector inefficiency, incorrect vector implantation and the possibility for a dangerous immune system reaction. In addition to these disadvantages,
At present, there are major disadvantages to gene therapy. Firstly, it is difficult to target specific cells. However, researchers are continually developing more efficient vectors such as virosomes. Secondly, gene therapy can cause dangerous immune responses, as seen in the case of Jesse Gelsinger. However, researchers have begun to use vectors that don't cause an immune response; such as adeno associated viruses, virosomes and liposomes. Scientists have also harvested cells and performed gene therapy outside the body (ex vivo). This method of gene therapy does not cause an immune response and scientists can check whether or not the functional gene has been implanted into the genome. In future, it is likely that ex vivo gene therapy will become more common, as it is safer than in Vivo gene therapy. Thirdly, for gene therapy to succeed, the functional gene must integrate into the genome, in the right location (on the end of a chromosome). vectors such as the adenovirus do not integrate into the genome permanently; and retroviruses implant into random parts of the genome, causing frameshift mutations (as seen in the gene therapy-leukemia incident). Scientists are continually investigating the use of safer vectors, such as adeno associated viruses, liposomes and virosomes. Finally, it is important to note that gene therapy currently has limited uses. Disorders that involve multiple genes, chromosome mutations and/or environmental factors cannot be treated with gene therapy. In light of the aforementioned evidence, it is apparent that gene therapy is currently dangerous and ineffective. However, gene therapy is an experimental technology which has only been trialled in humans since 1990. Therefore, it is too early to rule out the use of gene therapy for genetic conditions such as