The Pros And Cons Of Gene Therapy

Gene therapy was introduced into the scientific field around 1972, which led to the first attempt at its use in 1980. Although original efforts were unsuccessful, this event ignited research into the idea, which has led to the multiple developments in gene therapy still being refined today. Around the same time, biotechnology also emerged as a new and ingenious tool in field of biology which only furthered the potential abilities of gene therapy. Current research surrounding these fields combines biotechnology with gene therapy in utilizing viral vectors to incorporate targeted changes to DNA inside mutated or diseased cells. This method is hoped to eventually be used for treatment and prevention of incurable diseases such as Severe Combined
Currently, there are about 10,000 diseases that have been found to result from a mutation in precisely one gene out of the entire human genome (9). Fortunately, gene therapy is a relatively new scientific approach to treat and prevent diseases caused mainly by a single mutated gene, as well as those diseases that currently have no other treatment or cure (8). Gene therapy, which frequently coincides with biotechnology, emerged between the 1970s-80s when the company, Genentech, used bacteria to develop insulin that was genetically engineered to treat diabetes in humans (9). With this ingenious discovery, it was not surprising that scientists began using their biotechnology cohorts to hypothesize concepts of repairing flawed DNA with a more direct approach; as opposed to using prescription drugs or surgery (8,9). In 1989, it is documented that William Anderson became the first scientist to utilize a direct gene transfer technique in seeking to assess the safety of gene alterations by inserting a marker gene into DNA (9). In the decades following, there was a rapid influx of clinical trials geared towards various diseases that employed biotechnology to find different methods of performing gene therapy (9). Since then, the possibilities
AAVs are beneficial to gene therapy due to their ability to integrate DNA into specific sites on chromosomes, give long term expression of the gene, carry genes of smaller size and have no toxic effects to the human body (5). In contrast, AVs are larger and can therefore carry larger genes which is helpful because many genes, especially ones that will be discussed, are large and difficult to fit inside a small virus. Other types of viral vectors have similar qualities and all, or a combination, have been utilized for diseases such as Cystic Fibrosis, Sickle Cell Anemia, Emphysema, HIV infection and multiple types of cancer as well as other rarer disorders (5). Along with gene replacement, there are forms of gene therapy that deal with inactivating a disease-causing gene or introducing a completely new gene into human cells to combat a disease, however those methods come with additional adverse effects (8). The idea of gene therapy has gone from merely a concept to clinical trials in all stages, and although it may still come with some issues, there have been an influx of achievements in the field that will help to find future cures for incurable diseases