Copyright 1996


Gene therapy is thought of by some people as an terrific discovery that could greatly improve the human population and also give people with certain disabilities some glimmer of hope. And on the other side of the coin there is some people that think we have no business playing God. They feel that playing God may possibly cause devastating effects to the human gene pool and may also led to misuse.

Merriam-Webster’s Collegiate Dictionary defines gene therapy as the insertion of normal or genetically altered genes into cells usually to replace defective genes especially in the treatment of genetic disorders. By using gene therapy, we can go to t he base of the disorder instead of using drugs to alleviate symptoms. Some diseases that have been treated include ADA Deficiency, familial hypercholesterolemia, cystic fibrosis, cancers that include melanoma, Neuroblastoma, brain tumors and AIDS (Nation al Cancer Institute,1993).

Technical Aspects

There are three methods used to deliver the genetically altered material. The first method includes either retroviruses or retrotransposons. Retroviruses are viruses that can transfer their own genetic information and also genetically alter the huma n gene. These viruses are unable to copy themselves but still pose a problem in altering protein synthesis when these retroviruses splice a patient’s cells. This is were retroviruses come into play. There are parts of DNA from a cell that “ can copy th emselves onto other sites in the cells genome “ (Glausisus,1996). One type of transposon is a yeast transposon called Ty3. This yeast transposon is still under research.

The second method involves blasting genes with a pressurized gun filled with helium (Glausisus,1996). The helium gun will fire very small gold bullets which will be coated with the genetically altered genes. This technique was done on mice that had tumors. The gold bullets coated with the altered genes were shot at the skin of the mice that surrounded the tumors. The cells that were successful in receiving the altered genes code for cytokines that activate immune cells (Glausisus,1996). By the ac tivation of the immune cells, they hope the tumors will decrease. This study was done at Northwestern University by Wen Sun and colleagues. They found out by shooting the mice three to five times a day, there was a significant decrease in the size of t he tumors and an increase life span for the treated mice compared to the control mice (Glausisus,1996). By using the gene bullets the genes are not permanently in the cells of the mice but the effects ware off in a few days to a few weeks. This may be a n advantage or a disadvantage.

The third method involves liposomes, hollow fat molecules in solution. This method is being researched by the Royal Brmpton Hospital in London headed by Natasha Caplen (Glauisisus.1996). She is using liposomes in experimentation with cystic fibr osis. Cystic fibrosis is a disease that is caused by a chloride ion build up in the respiratory tract which causes difficulty in breathing. By inhaling liposomes coated with genetically altered genes prevent the build up of chloride ion they saw a signi ficant decrease in chloride ion levels. This method does not pose the harmful side effects that retroviruses do.

The first disease that was approved for gene therapy was adenosine demeans deficiency or ADA. This is a very rare disorder. A normal ADA gene produces an enzyme that is called adenosine deaminase. A person with ADA deficiency does not produce this enzyme. Children who have this deadly disease are seriously prone to the most minor of illnesses. If ADA Deficiency is untreated. life for these kids is only a couple of years and replacement ADA is limited. ADA Deficiency is effected by one gene whi ch makes it an easy target for gene therapy experimentation (National Cancer Institute,1993). This same procedure is under development and research for AIDS.

Another major issue that attracts gene therapy attention is cancer. An experiment done at the University of California in LA led by Habib Fakhrai done studies on rats with tumors. They had sixteen rats of which eleven of them received the genetica lly altered material called 9L gliosarcoma. By altering the cancer cell, it blocks off the synthesis of a protein called TGF - beta. TGF-beta greatly decreases the quality of the immune system. After all was said and done the eleven rats that t receiv ed the altered 9L gliosarcoma cells were alive and the five that did not died (Seligman,1996).

There are many other diseases that may be cured by gene therapy of which include Rubinstein-taybi syndrome, partial epilepsy, cataracts, prostate cancer , male infertility, Alzheimer’s, schizophrenia, usher syndrome, and maternal acute fatty liver of pregnancy. All of these disorders had there genes identified in 1995 (Glausisus,1995).

The Pros and Cons of Gene Therapy

The ethics that surround the issue of gene therapy are very overwhelming. Some people think that we will get burnt trying to play God while others think this is a great advancement for man kind. One reason for unacceptance may be that gene therapy m ay be used for the enhancement or modification of human capabilities. If this were feasible, the standards of a normal human being would be changed. Even if some small countries experimented with gene therapy, could they develop unstoppable armies? If gene therapy was done to a certain extent could it alter the human gene pool for good? And also would this form of treatment be a luxury only for the rich which could very well make the rich, richer and make the poor, poorer. For just these reasons an d theories, all gene therapy experiments must receive approval from the Recombinant DNA Advisory Committee (RAC) and the National Institutes of Health,(NIH). After approval by these committees the trials must also receive approval from the U.S. Food and Drug Administration (National Cancer Institute,1993).

Many countries have been approved for trials in human gene therapy including USA, China, France, Italy, and the Netherlands. The publics opinion has a lot of influence on whether the trials get approved or not. So a survey was done by Darryl R.J. M acer on ethics surrounding gene therapy in many different counties including the USA, Japan, New Zealand, UK, France, Italy and Germany.

These surveys were done from August of 1991 to October of 1991. All surveys were as random as possible. The questionnaires were delivered by mail in Japan and were also done face-to-face to get a better response rate in some cases.

The survey found out that 74% of the Japanese thought that gene therapy was unacceptable. In the USA 42% said that changing genetic makeup was wrong while 52% said it was acceptable. In both New Zealand and Japan, scientists found gene therapy more acceptable tha n teachers, and teachers more accepting than the public. In the USA, 24% of the people interviewer said they knew nothing about genetic engineering, 39% said they have heard of it while 35% said they knew more than average. In Japan and New Zealand, 63 % of the people that were in the survey suggested that gene therapy was a form of playing God. Also 20% of the people said that there must be a form of control to deter misuse. An opinion poll done in Europe, included countries of UK, France, Italy, and Germany, included that one third thought it is ethical and the other one third thought it was unethical while the last one third were undecided. Some of the consequences that were cited in a 1986 poll included, difficulties in the control of the spread , health hazards, mutants, environmental contamination, because knew and unknown diseases and also cause antibiotic resistant strains (Macer,1992). Whether or not these theories are correct or no we must still investigate the possibilities.

It is unsure to say that the advantages out weigh the disadvantages but to some people it may be their only chance to live. Man kind is still in need of more information surrounding this topic. I do think gene therapy could be a wonderful thing in curing series diseases that would other wise led to death. But the costs of gene therapy would certainly be outrageous. Gene therapy could possibly be only for the rich and not the poor. The public needs to be more informed about the discoveries in gen e therapy to aid in research. If the public is more willing to accept this our scientists will discover more and better things. But just like anything else, anything not in moderation could be devastating. The possibility of misuse could led to a highe r standard of normal or could led to misuse in other forms. For just these reasons we definitely need RAC (Recombinant DNA Advisory Committee), NIH (National Institutes of Health) and the U.S. Food and Drug Administration. Without these committees the p ossibility of misuse will be greatly enhanced.


1) Glausisuz,J.(1996).The genes of 1995. 1995:The Year in Science 17,36-38.

2) Macer,D.R.J.Public Acceptance of Human Gene Therapy and Perceptions of Human Genetic Manipualtion.Obtained from the WWW 10/28/96:http//

3) Britannica On Line: gene therapy. Obtained from the WWW 10/23/96:

4) National Cancer Institute.Questions and Answers About Gene Therapy. Obtained from the WWW 10/23/96:

5) Seligman,J.(1996).Hope for genetic healing. Newsweek,April 15,64.

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