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Gene Therapy: For Whom Is It Necessary and When? Meg Kiley The Art and Science of Negotiation. ABRSTRACT

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Gene Therapy: For Whom Is It Necessary and When?

Meg Kiley

The Art and Science of Negotiation


Currently, in the world of medicine there is an ethical debate over whether gene therapy is a science that should be studied further in order to one day become a norm in society like for example, organ transplants. Gene therapy holds the possibility of curing, and even eliminating entirely, genetic disease such as cystic fibrosis. Yet some people fear that advancement in the technology could also turn in the frightening direction of Eugenics, leading to circumstances like those in 1930’s Germany when a program took place (Eugenic Sterilization Law of 1933) which sterilized those who had any sort of debilitating disease. Both sides of the debate have legitimate, objective reasons why gene therapy is a positive medical advancement for society or a nightmarish tool to be used for unethical purposes.


All of these people have one main interest in common. They all believe that gene therapy could be beneficial.

They all want to treat diseased patients well and work to find cures.

They all think gene therapy should be used to its potential for improving health.

The question they have for each other is when will the science be appropriate to use on humans and who those humans might be. The terminally ill? Sick babies? The genes of embryos? When the patient is left with no other hope? When the patient consents? When the doctor thinks it appropriate? When it offers the possibility of eradicating large scale diseases like AIDS, cancer, and heart disease? Should it be constrained to monogeneic diseases?

The sick and their loved ones want all the options for recovery open to them.

People who are looking ahead want to make sure that the regulations for gene therapy are not with out boundaries.

People who are liable want to make sure that gene therapy is not going to cause any harm (researchers and doctors are interested in their reputations).


-Standardize the review committees’ regulations

-Create a referendum for the people to vote on

-Create more gene therapy trials which do not require human patients

-Create definitive guidelines as to whom is in need of gene therapy

The History of Gene Therapy

Gene therapy was first established in the 1970s. During its beginnings it was referred to as “gene surgery”. In the 1980s genetic research became more popular, and by the 1990s, the Humane Genome Project, a research team which set out to map the entire Human Genome, was officially put in place. Through this genetic mapping, more and more of the genes that when mutated cause diseases were being discovered. These diseases ranged from intestinal cancer, to heart disease, to Alzeimers. The first person to receive gene therapy was a 4 year old girl whose body was not producing an enzyme detrimental to a functioning immune system called adenosine deaminase (ADA). After cells with the proper genetic code for producing ADA were injected into her system, the girl showed improvement. This break through occured in September of 1990.

Another case of gene therapy did not work out so well. Widely publicized in 1999, 18 year old Jessie Gelsinger’s death after an experimental gene therapy treatment at the University of Pennsylvania was a blow to the science’s reputation. This unfortunate event was unexpected, in fact Jessie was not even terminally ill. “Jesse Gelsinger was not sick before died. He suffered from ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder...” Even after this mistake, many still regard gene therapy as a hopeful, viable option that just needs further research and development.

As the research continues, more and more uses for gene therapy are being realized. Not only can the specific gene mutations of diseases be reversed, but DNA that codes for other proteins which battle a certain disease can be used. For example, in cancer patients a DNA code can be inserted that makes the cancer cells more likely to respond to treatment.

There are some who fear that gene therapy will lead down a frightening road. The past has shown that gene technology can be very controversial. Extreme occurances date back to when the Nazis created a law, the Eugenic sterilization Law of 1933, which required that people with disabilities (defined by the Nazis) were sterilized so that they could not reproduce. Today the FDA (Food and Drug Administration) and other committees are in place to regulate the scientific research that is funded by the government. There are many gene therapy research trials currently underway with human patients.


Gene therapy is named therapy for a reason. The concept is that of repair of damage, not improvement of a healthy subject. Someone who is healthy is not in need of therapy. Therefore, a definition of health needs to be established and agreed on in order for all the people involved to be in agreement.

People Involved

There are many people who believe gene therapy is something that should be utilized for those who need it, even as it in the process of being developed. They include…

-people who suffer from terminal or life altering diseases for which gene therapy may be a beneficial option

-the loved ones of these people

-doctors who seek the best treatments for their patients

-Drug companies who could distribute and profit from gene therapy products

There are many people who do not believe gene therapy is something that should be used until researched much more extensively with more government control. These people include…

-those who believe that gene therapy has the potential to cause harm to future generations if not dealt with properly now

-those who believe that gene therapy in its early stages is more harmful than beneficial

-those who have already been negatively affected with the use of gene therapy (such as Jesse Gelsinger and his family and friends)


Hunt, Katherine S. and Teresa G. Odle. “Recent Developments in Gene Transfer Research: risk and ethics.” Alliance for Human Research Protection. 10 Jan 2005. <>

Sunkavally, Naveen. “Biotech experts Debate Gene Therapy, Regulation.” The Tech . 13 March 2000. Massachusetts Institute of Technology. 6 January, 2006. <>

“a NewsHour with Jim Lehrer Transcript Online Focus Gene Therapy.” Online NewsHour. 2 February, 2000. McNeil/Lehrer Productions. 24 January,2006. <>

“Gene Therapy.” Human Genome Project Information. 18 Nov. 2005. 1 Jan. 2006. <>

Objective Criteria

-There are 4,200 diseases that result from abnormal or mutated genes.

-There are oversight committees which review gene therapy research:

The Food and Drug Administration (FDA)

The Office of Protection of Research Risks (OPPR)

The Institutional Review Boards (IRBs)

The Institutional Biosafety Committees (IBCs)

-In Scientific American biochemistry researchers often do not disclose their company affiliations.

-More than 4,000 patients have already taken part in gene therapy studies.