What is this “Gene Therapy”?

How does it work?

What is its significance in society?

How is technology used in this?

What ethical issues might this involve?

What are the potential benefits/problems of this?

What is Gene Therapy?

-a simple definition by Karen Chen

Gene therapy is a relatively new technique used to treat genetic disorders and diseases. The method is mainly used to correct defective genes that are responsible for the development of the disease. The most common practice of gene therapy is to replace a mutant allele* with a functional one.

*for definition, visit http://dictionary.reference.com/browse/allele

How Gene Therapy Works

In the majority of studies on gene therapy, an ‘abnormal’ (disease-causing) gene is replaced by a ‘normal’ one by having the therapeutic gene inserted into the target cells of a patient. This is done by using carrier molecules called vectors to deliver the ‘normal’ gene to where it’s needed. Believe it or not, the most commonly used vector is a virus. Not any old virus though, these viruses have been genetically altered to carry normal human DNA. Nowadays, viruses have evolved a way of taking their own genes and delivering them to human cells to cause illness. Scientists have been able to use this to their advantage however by using this ability of theirs and manipulating the virus genome to remove the disease causing genes and insert remedial ones. If, for example, we wanted to replace abnormal cells in the liver, we would infect the patient’s liver cells with the viral vector. The vector will then dump its genetic material containing the normal human gene into the target cell. With the new therapeutic gene, the cell will generate functional protein which restores the cell to a normal, healthy state. Now, would everyone please have a look at this website:

http://www.fda.gov/fdac/features/2000/gene.html

Notes for Mission

GTThis tape appears to

be a recording from

some sort of class on

Gene Therapy. I’ve

gotten my text gadget

to record the words

into a text version of

it for quick reference.

The clue was found at

10:46 AM, Thursday,

October 11th, 2007.

October 11th, 2007

Toronto-

It seems that everyday now a new biotechnology project comes in and you wish you had paid more attention in biology class.

At a press conference concerning recent developments in gene therapy research, a brave man dared to ask, “How is gene therapy significan to us?”

Dr. Peter Evans of the Gene Therapy Research Organization (GTRO) cleared his voice before answering, “Gene therapy is really an exciting topic. It can be used to do so much. Most commonly, we use it to treat genetic disorders like severe immunodeficiencies and so on. We’re expanding its uses all the time though, we have guinea pigs that were deaf with it, and we recently had scientists prove that gene therapy can be used to effectively treat cancer.”

“It can be used to do so much,” that quote sums it up pretty well. Upon further research, I saw the names of disorders and diseases treatable by gene therapy stacking up. Genetic disorders like X-SCID (severe combined immunodeficiency) or the "bubble boy" disease, sickle cell, myeloid blood diseases, even blindness were among a list of many others with results in varying success. Even Alzheimer's and weight loss made the list. For the future, gene therapy is looked at as a possible treatment for Parkinson’s and is already on its road to becoming an effective cure to some cancers. Ways of using gene therapy as a prevention technique against cancer and diabetes is also being looked into. If I were to bet my bottom dollar on how significant gene therapy is, I would; it’s just that significant.

Hmmm. Where could the next

clue be?

Actually, I am going to check my email, I deserve a

break.

To: 008 beachlover380@hotmail.com

From: 307 skyhigh432@yahoo.com

Subject: Technology and GT

Gene Therapy itself is considered a technology but Agent 000 told me to give you some info about the specifics on viral vectors and

Do they need to be cured or prevented?

Some say: Yes, life is simply much easier without having to deal with a disability and no one should have to go through life like that.

Others say: No, having a disability doesn’t make you any less human and you shouldn’t be regarded differently. Why should we try so hard to cure disabilities many people are living with today? It is practically demeaning their existence.

Some say: Everyone and anyone should have access to these therapies. We are all entitled to equal rights and should be able to have the therapy we need performed no matter who we are and how much money we have. The government should pay for our fees, or at least subsidize them. If a treatment is available, anyone should be able to go have it done, no matter their financial situation.

Others say: As much as we’d like to idealize and say that everyone should be able to have gene therapy performed, our society simply doesn’t work that way. It is extremely costly to have to procedure done and that kind of money is difficult to come by. There is almost no way that a patient could have it done for free without some serious changes like huge increases in funds or higher taxes in countries. A potential patient would have to be very financially secure for this treatment which unfortunately, at least for now, cannot be accessed by all.

Somatic gene therapy is performed on adults who are known to have the disease while germline therapy is done is egg and sperm cells to prevent a trait from being passed down from generation to generation.

I personally think that germline therapy is perfectly acceptable. Idealistically speaking, if we prevented traits from being passed down, we could eventually get rid of a disease altogether. It also feels more safe knowing that you won’t inherit a disorder from your parents or that you won’t pass one on to your children. On the other hand, it is also unfair to determine something for others without letting them have the chance to make a decision for themselves. This would make somatic gene therapy more ‘fair’ since it is your personal decision but when we are born, we also have no hand in deciding what genes we want.