Can Gene Therapy Work – Part I

The subject of Gene Therapy comes up quite often. It is an interesting concept and a field of study that is making tremendous strides in the medical and research communities today. This is a two-part article on the subject. Part I will discuss the general subject of gene therapy including the pros and cons as well as the ethics of this therapy. Part II will focus on the possible application of gene therapy as a treatment for Kennedy's Disease.

Definition and Background

Wikipedia defines Gene Therapy as "the insertion of genes into an individual's cell and biological tissues to treat disease, such as cancer where deleterious mutant alleles are replaced with functional ones. Although the technology is still in its infancy, it has been used with some success. Scientific breakthroughs continue to move gene therapy toward mainstream medicine."

"Today, most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. The biology of human gene therapy remains complex and many techniques need further development. Many diseases and their strict genetic link need to be understood more fully before gene therapy can be used appropriately. The public policy debate surrounding the possible use of genetically engineered material in human subjects has been equally complex. Major participants in the debate have come from the fields of biology, government, law, medicine, philosophy, politics, and religion, each bringing different views to the discussion." [Wikipedia also includes a well-written section titled "Problems and Ethics."]

Buzzle.com states "Gene therapy can be broadly classified into two types. One is the somatic cell gene therapy and the other is the reproductive cell or germline gene therapy. In somatic cell gene therapy, the somatic cells are targeted for gene replacement, whereas in the reproductive cell gene therapy, the defective gene lies in the reproductive cells that are replaced by the correct gene."

[Buzzle also provides some Pros and Cons on this type therapy.] "Gene Therapy Pros - The single most important factor that gives gene therapy its edge is its incredible therapeutic potential. The human race has always been under the continuous onslaught of diseases. As we find a cure for some of the diseases, new and more virulent forms of germs attack us. Although such diseases can be cured through medicines, genetic disorders have no cure unless the defective gene is replaced by the correct one and this is what gene therapy aims at. Moreover, by targeting the reproductive cells, such defects can be got rid of for good. People suffering from genetic disorders like Parkinson's disease, Alzheimer's disease and Huntington's disease are some of those whose only hope for cure is gene therapy."

"Gene Therapy Cons - Scientific Issues: Although the most popular choice as a vector, using virus for the purpose is not foolproof. There is a specific point within the host genome where the correct gene should be introduced. There is no guarantee that the viral enzyme that is responsible for this step will be able to introduce the correct gene at the specific point in the host chromosome. Moreover, the body's immune system may destroy the vector as it may perceive the carrier as a foreign body. Then there are problems with introducing therapeutic DNA and the rapidly dividing nature of certain cells that are hurdles in gene therapy providing long-term benefits to patients. Due to this reason, a patient may need to undergo multiple gene therapy treatments. This again focuses on the problem of the immune system. Once the immune system is triggered by a foreign body, it attacks the foreign body more aggressively when it invades the body next time.

Ethical Issues: Given the technology involved, it is obvious that gene therapy treatment will be expensive. It will be just the rich who would be able to afford its benefits. We know the potential of reproductive gene therapy. The scope of this line of treatment triggers the fear of eugenics - a term that denotes creation of a superior race, the idea that media has tried to embody in the concept of designer babies. Although this definitely isn't on the minds of geneticists but it is difficult to erase the repercussions of Hitler's belief in supremacy of the Aryan race.

Religious Issues: Manipulating genetic makeup of man is absolutely unacceptable by those with strong religious beliefs. According to them altering genes is similar to tinkering with nature. It's like questioning God's will or in other words, 'playing God'."

I found the topic of Gene Therapy interesting. I definitely had some assumptions on the subject, but found that there is much more to the story than originally thought. Getting beyond the issues and ethics questions will not happen overnight. When you look at the subject of abortion and consider how volatile that issue is today (37 years after Rowe vs. Wade); it makes you wonder whether gene therapy will develop into a similar controversy.

As mentioned in the lead-in, Part II of this article will discuss the potential application of gene therapy as a remedy for Kennedy's Disease. As they say, "that is where the rubber meets the road."