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Gene Therapy

Gene therapy is an innovative method of treating diseases by changing the makeup of an individual’s genes toward a beneficial end. Gene therapy is most often looked at as a way to cure debilitating and deadly diseases but it also has the possibility of preventing disease in the first place. There is promise with each day and each new discovery as more successful evidence is found. Nonetheless, gene therapy remains a very tentative science whose possibilities are still yet to be fully realized.

You know how you always wanted to blame your parents for your mistakes? Well now you can. Your entire structure is made up of your parent’s chromosomes, XX if you’re a girl and XY if you’re a boy. The reason for genetic disease is from defective gene traits. In the majority of cases, one regular gene is enough to live normally. If the defective gene is recessive, then the dominant gene will carry on both tasks. If two recessive defective genes are present at birth then the disease becomes apparent. If the defective gene is dominant though, then it will carry on the task of both genes, even if the other is normal. Therefore, only children with a parent who has the disease may be affected by it. Males have one X and one Y chromosome, the X from the mother and he Y from the father, therefore each cell has only one copy of the genes on these chromosomes. There can be sex-linked diseases which can be carried by mothers and given to their sons, all who lack two X chromosomes. Essentially, because there is no other gene to counteract the defective one, the male inherits the disease. A perfect example of this is hemophilia.

Hemophiliacs lack certain protein enzymes in their blood supply. Affects of this disease are that individuals are not able to produce blood clots. Therefore, a cut or bruise that would seem normal to the average person, might be deadly to a hemophiliac. In former years this was treated by using other individuals blood to extract the enzymes. A serious problem occurred when the AIDS virus tainted some of the blood supply. The use of gene therapy could have prevented this tragedy from taking place.

The basic idea of gene therapy is based on correcting genes by manipulating their DNA. There are 2 types of gene therapy, the first is somatic gene therapy. Somatic gene therapy involves the manipulation of genes in an individuals cells so that it cures the patient, but this alteration is not inherited by the individuals children. This is the type of gene therapy that is currently put into practice in laboratories around the world. The other form is called germinal gene therapy. Germinal gene therapy is where the germ line cells are set so that the individual as well as his or her offspring will have the corrected genes. Because of ethical issues, germinal gene therapy is only practiced on animals. It is not expected to be performed on humans anytime soon.

It all seems nice and peachy, but there are problems with gene therapy. Finding a reliable way to get the corrected genes into the body is proving to be quite a lofty task. There are two main ways in getting the corrected genes into the body – retrovirus and adenovirus. Retroviruses are a class of enveloped viruses containing a single stranded RNA molecule as the genome. Adenoviruses are non-enveloped viruses containing a linear double stranded DNA genome. Both techniques are quite successful but do have their drawbacks. Retroviruses for example can mutate once inside the body and can even cause cancer or can disrupt other genes. Adenoviruses are not able to mutate within the body, but they are not as stable as retroviruses and are harder to manipulate because they are selective to mainly the lungs Another problem with using viruses is that the body’s natural immune system will compete against these viruses. These viruses are still in their clinical stages and will hopefully be perfected soon.

There is also always a chance that something might go wrong when dealing with DNA and viruses. Safety becomes an issue when dealing with all these biological materials. A virus might escape and get out into the general population or contaminate something. A lab worker may prick himself on a needle and thus gain an undesirable effect. Then again things can go wrong in anything, not only in solely gene therapy. A man could be crossing the road and get struck by a car. There is always a risk when dealing with anything, so why walk out of the house?

Will gene therapy be offered the general public? I’m sure not right away in it’s early stages; gene therapy is quite costly and is still experimental. Though, we can always count on the trusty ole government to pass laws pertaining to the practice of gene therapy. In the future don’t be surprised if gene therapy is included in your HMO. The government controls and regulates health care in this country. They control doctors’ pay and insurance companies’ coverage.

Another problem that might arise would most likely be covered by the government as well. Who is to say what is a disease and what is not? One is likely to say that being short or being brunette is just as much a disadvantage as to have diabetes. People will want to change their appearance with the use of gene therapy. The government will step in here and outline what is and what is not considered a disease. Just like with what is covered by insurance companies, a triple by-pass is covered under many insurances while I think one would have to look extensively to find an insurance company who covers a nose job. Everything is to be taken into moderation.

Of course there are people who always ask about the “long run,” like what about world domination and overcrowding? World domination would never happen this day in age. First of all there you would have to have a dictatorship and a small enough country which could be manipulated into complying with these laws. Second, there is a very slim chance that a small country could hide the fact that they are practicing gene therapy for malevolent purposes, from larger superpowers like the United States and the United Nations. Then overcrowding won’t be an issue because just like with anything when one solution is discovered five more show up. With new technological advances though these problems can be solved and answers utilized. Higher buildings can be built and the use of Genetically modified foods are just two solutions.

The benefits of gene therapy greatly outweigh the risk involved. Not only can single variable traits be altered but such diverse diseases as cancer and AIDS may be cured. It is very encouraging to see that advances in such important fields of research are being made. Ethical issues are not even worried about given that germinal gene therapy is not in practice on humans. There is so much that can be solved with the use of gene therapy. What is the use of knowledge, if it is not applied?

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