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History And Procedures of Gene Therapy

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The History And Procedures of Gene Therapy


Abstract: Over the course of history there has been the idea of gene therapy has inspired many great scientists. The history of eugenics is important to the history of gene therapy because it is how gene therapy originated. Eugenics has driven many people to take extreme measures to try and make a “better human race”, this includes the Nazi party and the movement in the 1930’s inspired by Francis Galton. After that, research in eugenics continued and the human genome project sprung from the minds of scientists. After the inspiration of the human genome project scientists developed theories that gene therapy is possible. After a series of experiments that then failed, scientists were discouraged and the future of gene therapy is now being doubted.
Gene therapy is an idea that has fascinated humans and scientists for centuries. Gene therapy theoretically can allow scientists to change the human genome and peoples DNA. Ideally, scientists wish to replace “bad genes” in your DNA that can potentially hurt you with good genes that will help you avoid any genetic ailment. In fact, all that scientists really want to accomplish is to replace our natural genes with unnatural ones. The idea and concept of gene therapy is not new to human history. On the contrary, gene therapy is a concept that has been fascinating the minds of many scientists and researchers over the span of many centuries.
Although the idea of genes was first born during the times of Plato, the concept of gene therapy wasn’t really born until the late 1900’s. Mr. Francis Galton, who is the younger first cousin of Charles Darwin, had the idea that the human race could be improved according to the method of selective breeding. This idea came about by first, discovering that genes determine which characteristics you have in your appearance and behavior. Gregor Mendel first discovered this concept during his pea experiments in the late 1800’s. After the laws of dominant and recessive genes were established after Mendel’s experiments, scientists were testing on fruit flies to continue research on genes.
After a while, the study of genes was given a name, eugenics. The idea became popular among the upper class and middle-class whites in countries such as the United States, Britain, and Germany. These people wanted to rid their society of “blight” by setting up a sort of breeding program that would exclude genes that could cause violent behaviors and conditions such as alcoholism, insanity, epilepsy etc. Scientists also wanted to eliminate genes and DNA that coded for genetic diseases such as diabetes, tuberculosis, and heart problems.
During the reign of Adolph Hitler, eugenics was used for less than good intentions, the Nazi party wished to use eugenics for “the betterment of the human race”. The Nazi party took over the eugenics labs and funded them to research genes that coded for things such as intelligence, behavior and ethnicity. The Nazi’s hoped to use this research to eliminate color and make just one completely Caucasian race. In their mind, the betterment of the human race was a civilization made up of all white people and no color of any kind. Needless to say that after the reign of Hitler, eugenics was given a very bad name and was often associated with a practice that was not quite humane and in fact cruel.
After World War II, research in eugenics and gene therapy continued throughout the decades. In 1973, a great advance in eugenics occurred, the creation and the invention of recombinant DNA appeared. DNA is the material in an organism that regulates how that organisms body functions. DNA is made up of amino acids, sugars and phosphates. The amino acids are A, T, C, and, G (A for Adenine, T for Thymine, C for cytosine and G for Guanine). These amino acids can only be fitted in pairs so A is paired with T and C is paired with G which then form a code. When these base pairs are put in a specific
sequence they code for a specific protein, which causes some kind of reaction in the body because the proteins call for enzymes. Recombinant DNA is the method in which a piece of DNA can be taken out of a genome and recombined with another. Therefore, it is the bases of gene therapy, which is taking a piece of DNA and either replacing, inserting, or extracting another piece of DNA. The exact procedure for recombinant DNA requires proteins that are also called restriction enzymes that bind and cut DNA in a specific place with another sequence of base pairs.
In the mid-1980’s, there were two scientists named Robert Sinsheimer and Charles DeLisis. Robert Sinsheimer was a successful microbiologist who partnered up with Charles DeLisis. Charles was a successful physicist by training and a former chief in mathematical biology. “He was intent on researching the effects of radiation and who it affected, especially what effects it had on mutating DNA. One day, as Charles was reading a draft paper, he had a revelation; he could compare the genome of a child to that of an adult to analyze the mutations that radiation caused” (Kevles pg.19). From there sprung the idea of the human genome project and along with the help of a lot of scientists and funding, the project was underway.
In 1986, DeLisis made a plan for a five year DOE human genome program that would include mapping, and the development of automated high-speed sequencing technologies, and research into the computer analysis of sequencing data. Before the human genome project can really be pursued, technology first had to be invented to be able to read the DNA and translate it so that scientists can understand what the DNA is coding for and how it affects the organism during life. After the first human genome research labs were established in the United States other countries caught on as well. For
example, several countries in Europe were gathering momentum researching the human genome during 1987-1988. Overall, scientists were convinced that the human genome project was the key to understanding life.
There is great relevance between the human genome project and gene therapy, because the concept of gene therapy sprung out of the human genome project. The human genome project was originally established to analyze the human DNA and understand what the DNA codes for and what results from that coding. This can mean that the DNA can code for brown hair, blue eyes, or hemophilia. After researching the human genome project, scientists found that they could add, replace, and remove etc. parts of the DNA. Hence, this is how gene therapy started, and it is basically the history of gene therapy. After the realization that the idea of gene therapy was slightly possible, scientists were researching how to carry out the procedures of gene therapy and then started to experiment.
When gene therapy was first discovered to be possible various scientists and the public considered it the miracle cure. “They considered gene therapy to be the answer to all sorts of genetic diseases and the cure to death causing ailments such as severe immune deficiency” ( Weiss pg 45). Although scientists have done many experiments hoping for success, there has been a considerable amount of disappointment occurring about the progress that has been made in using gene therapy for medical reasons. Over the past years there have been numerous experiments that scientists have carried out by using gene therapy on test subjects. Of course, with the approval of the FDA, scientists were researching monkeys and using them to experiment on DNA. In fact, in the year 1999 a monkey was used to experiment gene therapy. The monkey then died of cancer six years
later in the year 2005. This is not the only case of failure in the animals that were used in the gene therapy experiments, there have been various outcomes from the past experiments.
In Europe there have been several attempts to treat patients ailments by using gene therapy. Many of these attempts have failed that have also caused doubt among scientists as to the actual success that gene therapy will have. The only successful cases are of 12 patients who were all children in Europe who had a severe immune system deficiency. If this were not treated the children would be exposed to thousands of bacteria and, because of the lack of immune system competence, would then receive all sorts of infections and most likely die in the first stages of their life. Everyone was quite relieved that gene therapy seemed to solve this problem and the children seemed to be healthy enough after the therapy. Unfortunately, three of the children who were treated using gene therapy have now developed cancer and one child has actually died from it. Needless to say, this information was quite discouraging to scientists and researchers alike. In the year 2005, the FDA declared that gene therapy was too dangerous a field to pursue without extensive security and safety issues.
In conclusion, there is a great amount of history that goes along with the subject of gene therapy that goes all the way from Plato, to the human genome project. Gene therapy is a subject that has been misunderstood and even given a bad name. This has been caused by the mistreatment of gene therapy by such parties as the Nazi party during World War II and the Holocaust. Curiosity on the subject of eugenics and DNA then led up to the human genome project in which it was discovered that gene therapy is theoretically possible to carry out. Gene therapy was a beacon of light to a generation of
people that believed that it was the cure to never getting sick again and the ultimate medical treatment. But of course, there has been some discouraging outcomes and disappointment considering the subject but people still hope that one day, gene therapy is going to be the answer to disease resistance.


Bibliography
“Boy’s Cancer Prompts FDA to Halt Gene Therapy”
Weiss, Rick Washington Post March 4, 2005 pg A02
“Gene Therapy for Immune Disorders: Good News tempered by Bad News”
Puck, Jennifer & Malech, Harry. National Human Genome Research institute. Genetics and Molecular Biology Branch. January 31, 2006. Accessed June 17, 2007. http://www.sciencedirect.com/science?_ob=ArticleURL&_udi=B6WH4-4JS81T0-V&_coverDate=04%2F30%2F2006&_alid=425455301&_rdoc=1&_fmt=&_orig=search&_qd=1&_cdi=6840&_sort=d&view=c&_acct=C000059598&_version=1&_urlVersion=0&_userid=4421&md5=a695c90c485f3dd3a572bcbe42604583
“The Last 80 Years in Primary Immunodeficiency: How Far Have We Come, How Far Need We Go” Shearer, William & Fischer, Alain Department of Pediatrics and Immunology, Baylor College of Medicine, and the Department of Allergy and Immunology. February 9, 2006. Accessed June 17. http://www.sciencedirect.com/science?_ob=ArticleURL&_udi=B6WH4-4JS81T0-M&_coverDate=04%2F30%2F2006&_alid=425456057&_rdoc=1&_fmt=&_orig=search&_qd=1&_cdi=6840&_sort=d&view=c&_acct=C000059598&_version=1&_urlVersion=0&_userid=4421&md5=b273338ae3c563bfa35380a2e42aff41
United States. Environmental Protection Agency. Office of
Emergency and Remedial Response. This Is Superfund .
Jan. 2000. 16 Aug. 2002 superfund/whatissf/sfguide.htm>.
Lyon, Jeff & Corner, Peter. Altered Fates: Gene Therapy and the Retooling of Human Life. W. W. Norton & Company Inc. New York, New York. P. 1995.

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