Human Gene Therapy

Human Gene Therapy

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As the world gets older, there are more and more diseases. Not every illness has a cure. But there are some scientific advances that can help with these illnesses. One of those, hopefully, will be the human gene therapy. A gene has nucleotide sequences, that when disrupted could create problems with protein synthesis; thus, diseases develop (Sandhyarani, 2009). What human gene therapy is, is inserting a nucleotide sequence into an individual’s gene, replacing the defective allele. Cell and tissue function would then continue on normally, and the disease would disappear.
Viruses are used as a way for the therapeutic gene to be inserted. They act as vectors that carry the new gene into the cell (Sandhyarani, 2009). Though there are risks when using viruses. The viruses can cause inflammation problems in the immune system and organ failure (Mayo Clinic, 2010). There is also always the possibility that the viruses could cause cancer and tumors (Mayo Clinic, 2010). Other risks involved with the human gene therapy include misguided insertions of the therapeutic gene and overproduction of a protein (Risk Factors, 2011). There is still a lot of research to be done so all these risks can disappear.
One of the diseases the human gene therapy might be able to treat would be Severe Combined Immune Deficiency (ASGCT, 2011). This disease is when children are born with a dysfunctional immune system. These victims are prone to infections and need bone marrow transplant. Six patients in Italy were treated using the human gene therapy (ASGCT, 2011). A therapeutic gene was inserted into the bone marrow cells. The patients were able to live their lives normally, with no side effects (ASGCT, 2011). Another disease the human gene therapy has been able to help in is Chronic Granulomatus Disorder (ASGCT, 2011). It is another immune system disease. Two patients in Germany whose bodies were not able to fight off infections are now living normal lives as they were treated with the human gene therapy (ASGCT, 2011).
In the United States, the human gene therapy was tested for hemophilia (ASGCT, 2011). This is a condition where internal bleeding occurs because the blood cannot clot, sometimes causing death. Although the therapeutic gene inserted helped in creating blood clots, there were still problems with the immune system rejecting it (ASGCT, 2011). The most popular disease being tested with human gene therapy is cancer. Of the trials with human gene therapy, two-thirds have to do with cancer (ASGCT, 2011).

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Other diseases worth noting that are also being tested include HIV, diabetes, and Parkinson’s disease (ASGCT, 2011).
People have brought up a lot of ethical issues involved with the human gene therapy. One of them having to do with the idea of “designer” children (Ruggles, 1996). This is the concept that parents can choose desirable traits for their children (Ruggles, 1996). Many believe it is not right for parents to be able to design their children. To some religions, it is taking the role of God (Ruggles, 1996). Another issue has to do with who owns the information from all the gene therapy research. Most of the research was paid using federal funds; thus, the American society believes they do not have to pay for the information (Ruggles, 1996). Companies who only care about the money they can make off of the human gene therapy have already patented genes (Ruggles, 1996). As it is still not safe to use gene therapy, these issues are pushed back.
A unique question I have that the human gene therapy could help answer is, “What can aid in thwarting lupus?” Lupus is one of the many diseases where human gene therapy research has been done. With the research, scientists have learned which gene is the one that stops the overproduction of autoantibodies (Minerd, 2005). Some experiments were done with mice. With these experiments, the scientists were able to see that gene therapy could be used in the future for lupus. Some of the mice were given gene therapy, while others had no treatment. All the mice were inclined to autoimmune diseases (Minerd, 2005). The mice with no treatment developed some sort of autoimmune disease, and died (Minerd, 2005). The group of mice treated with the gene therapy had lower levels of autoantibodies and did not have renal failure, which is the leading cause of death in lupus (Minerd, 2005). Lupus is a disease that is hard to diagnose and people might be too late when learning that they have it. These incredible findings might be able to help doctors cure lupus or prevent it.
Human gene therapy has the potential to be a huge success. For right now, there are too many risk factors for it to be used on people. But human gene therapy might be able to be the cure that so many people desperately need. It could one day help save millions of lives. All that is needed is more time and research.



Works Cited

ASGCT (American Society of Gene and Cell Therapy). (2011). Retrieved from http://www.asgct.org/about_gene_therapy/diseases.php
Mayo Clinic. (2010). Retrieved from http://www.mayoclinic.com/health/gene-therapy/MY00105/DSECTION=risks
Minerd, J. (2005). Retrieved from http://www.medpagetoday.com/Genetics/GeneralGenetics/403
Risk factors. (2011). Retrieved from http://www.genetherapynet.com/viral-vectors/risk-factors.html
Ruggles, A. (1996). Retrieved from http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/
amanda.htm
Sandhyarani, N. (2009). Retrieved from http://www.buzzle.com/articles/what-is-human-gene-therapy.html

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