Phil347N: Critical Thinking - Argumentative Paper: Gene Therapy

SCID, hemophilia, muscular dystrophy, cystic fibrosis, and certain cancers are just a few genetic diseases that children can be born with. While some of these diseases is not a death sentence to those children, they li ...

SCID, hemophilia, muscular dystrophy, cystic fibrosis, and certain cancers are just a few genetic diseases that children can be born with. While some of these diseases is not a death sentence to those children, they live with a severely reduced quality of life, there are some that are a death sentence to those born with it. There is hope however, in the last two decades there have been major scientific advancements in the field of gene therapy. Imagine, taking down the disease before it can even begin to hurt an unlucky child or treat a suffering patient and cure him of the disease. Scientists have found if the mutated genes that cause the disease were to be changed back to their normal forms or replaced with a working gene, the disease would not manifest itself. This was tested in 1990 with the first gene therapy trial in the US, to treat a little girl who has SCID and was found to work temporarily. SCID is short for severe combined immunodeficiency and the children that are born with it are born without a working immune system [9]. Since that test in 1990 there have been multiple successes in treating SCID, culminating in the announcement in November 2013 that there had been five children who were successfully treated with gene therapy and were doing well [3]. With these successes, gene therapy was well on its way to becoming front page news, but public perception of the fastgrowing science was negative as movies and books painted it as a eugenics movement if research for gene therapy continued. This sparked a hard look into the ethics of gene therapy and the ramifications that it could have on society. As time passed and new technology and techniques were researched, more information was released and explained to the public showing the growing field in a positive light and removing the fears and stigma associated with gene therapy. Use of Vectors This study source was downloaded by 100000823250788 from CourseHero.com on 05-09-2021 13:14:04 GMT -05:00 https://www.coursehero.com/file/62484433/Argumentative-Paper-Finaldocx/ This study resource was shared via CourseHero.com Gene Therapy 3 According to dictionary.com gene therapy is, “the application of genetic engineering to the transplantation of genes into human cells in order to cure a disease caused by a genetic defect, as a missing enzyme” [2]. There are two major ways this can be accomplished, viral and non-viral vectors. Non-viral vectors are biomolecules such as liposomes and have been showing promise in some trials. They are easy to prepare, there is no limit in size of genes that can be inserted into it, and they have much less chance of causing an immunogenic response [5]. There are downsides to non-viral vectors, they have a hard time targeting specific cells and are inefficient in gene transfer to the nucleus. Viral vectors are very promising in this regard because viruses have been evolving for millions of years to transfer genetic material into its target. There are many different viral choices, each with their own strengths and weaknesses, such as retroviruses, adenoviruses, and adeno associated viruses to name a few [5]. Using viruses may seem counter intuitive to treat a disease, but they are prepared in specific ways that remove all harmful viral genes and makes them safe for use. There are cons to using viral vectors, they have a higher chance of immunological responses than biomolecules and can have toxicity. These problems have been studied extensively and can be addressed on a case by case basis. Because of these issues’ scientists have been experimenting with combining viral vectors in order to create a perfect vector. One that, in theory, “be able to be administered by non-invasive delivery routes, target to the desired number of cells within target tissue, and express a therapeutic amount of transgene product with the desired regulation for a defined length of time” [4]