Is it ethical to use gene therapy to cure genetic disorders?

1. Is it ethical to use gene therapy to cure genetic disorders? Courtesy of slimfilms.com Mercer University School of Engineering EGR 108 Professional Practices Dr. Marjorie Davis J. Ryans

2. The Bubble Boy • In 2002, then 18-month-old Rhys Evans received one of the first successful gene therapy treatments for a genetic disorder, which cured his severe combined immunodeficiency (SCID). Courtesy of news.bbc.co.uk

3. Common Terminology • Adenovirus- Originally a harmful virus filled with double-stranded DNA that can be modified into a transport vessel for inserting therapeutic genes. • DNA- A very long molecule that carries a cell's genetic information. • Gene- A segment of DNA found on a chromosome that codes for a particular protein. • Genome- The sum of all genes that code for a particular organism. • Vector- Gene therapy delivery vehicles, or carriers, that encapsulate therapeutic genes for delivery to cells. These include both genetically disabled viruses such as adenovirus and non-viral vectors such as liposomes.

4. The Gene Therapy Process 1 2 A desired gene is selected and inserted into a genome to replace an “abnormal”, disease-causing gene. The genome is then transferred into a vector, most commonly an adenovirus, which will transport the gene into the patient. The vector is sent to a target cell in the patient’s genome and then “unloads” the genetic material in the virus. Once the genetic material has been inserted into the target cell, the cell will continue its reproductive cycle with the new therapeutic gene . This will cause new generations of cells with the integral therapeutic gene which will cure the desired disease in the patient. 4 3

5. Application of Gene Therapy One of the primary uses for gene therapy at its current stage is to cure particular genetic disorders. These include hemophilia, chronic granulomatus disorder, and severe combined immune deficiency. In the future gene therapy will hopefully be able to cure other types of acquired diseases. Such as neurodegenerative diseases, hepatitis, and even cancer. Courtesy of news.bbc.co.uk

6. Major Advancements • 2002- Scientists are able cure children of SCID. • 2003- Researchers are able to insert genes to the brain, opening up a wider variety of diseases that could be cured. • 2006- Scientists successfully treat two cancer patients.

7. Problems with Gene Therapy • Difficult to integrate desired DNA into a genome. • Effects are usually temporary so patients need multiple rounds of treatment. • Immune system automatically attacks foreign objects in the body, which can reduce effects of gene therapy. • The possibility of the modified adenovirus to revert back to its harmful state. • In rare cases therapy has induced tumors, specifically leukemia.

8. Economics of Gene Therapy • Funding for therapy fluctuates with rise and fall of success. • Most funding in government research, averaging $400 million a year. • Extremely difficult for most of society to afford and can’t be covered by insurance. Courtesy of dir.nichd.nih.gov

9. Ethical Issues • Who decides what diseases to cure? • Who will have access to therapy? • Is it interfering with God’s plan? • Will it bring about negative eugenics? • Is it alright to use the therapy in the prenatal stage of development in babies? Courtesy of www.cbs.dtu.dk

10. Conclusion • Gene therapy has the capability of curing numerous, common diseases in the future. • Therapy can also have severe, unforeseen circumstances. • At its current stage, therapy not accessible to most of society due to cost. • The ethical issues of gene therapy greatly slow its progress, but most are important to consider before expanding the development of the technology.

11. Annotated Bibliography • Baum, C., Schambach, A., Modlich, U., & Thrasher, A. (2007). [Gene therapy of SCID-X1]. Medline, RetrievedMarch 15, 2008, from http://www.ncbi.nlm.nih.gov/pubmed/18046520?ordinalpos=13&itool=EntrezSystem2.PEntrez. Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSum • This source was an article that gave technical insights to how a common genetic disease works and ways for gene therapy to combat it. It’s also a highly reliable source because the paper come from a top government research institute. • Maugh, Thomas (2007). Gene Therapy. In Microsoft® Encarta® Online Encyclopedia [Web]. Microsoft Corporation. Retrieved March 15, 2008, from http://encarta.msn.com/encyclopedia_761579447_3/Gene_Therapy.html • This source provided mostly general information of how gene therapy works and the possible applications of it. This source also provided very good media components to help convey the idea of gene therapy. • Terminology. Retrieved March 15, 2008, from American Society of Gene Therapy Web site: http://www.asgt.org/about_gene_therapy/terminology.php • This source was helpful in providing terms I could tell my audience prior to explaining gene therapy. For such a technical subject it would be almost impossible to accurately explain the concept without knowing certain terms. • Hoag, H (2005, May, 26). Gene therapy rising? Nature, 435, Retrieved March 15, 2008, from http://www.nature.com/nature/journal/v435/n7041/full/nj7041-530a.html • This article gives opinions and statistics about the economics of gene therapy. This is important because it gives possible options for people in the future for accessing gene therapy and who it will be accessible to.