Advancements in Gene Therapy

By: Liz Acque

Gene Therapy. What is it?

It is an experimental technique that uses genes to treat or prevent diseases. Researchers are testing approaches to gene therapy like replacing a mutated, disease causing gene with a healthy gene, inactivating a mutated gene that is functioning improperly, and introducing a new gene into a body to fight a disease. It is known to be a risky technique and is still in the process of undergoing studies to assure that it is both safe and effective. It is currently being tested on diseases that do not have a cure.

The Magic Behind Gene Therapy

Currently very few people have received completely successful gene therapy. Although there haven’t been very successful stats so far, the future of gene therapy looks very bright and promising. Decades of research have taught us a lot about designing safe vectors, targeting varieties of cells and managing and minimizing immune responses. Many clinical trials are underway to insure that once gene therapy is brought to the clinics that it is both safe and effective for everyone. In the future gene therapy has the potential to completely reverse, cure or reduce symptoms of immune deficiencies, hereditary blindness, hemophilia, blood disease, fat metabolism disorder, some forms of cancer and Parkinson’s disease.

How Does Gene Therapy Work?

Advantages and Disadvantages

The advantages of these treatments definitely outweigh the disadvantages. These treatments can give someone born with a genetic disorder the chance to have a normal life and will also eradicate the chances of their offspring being affected by the disorder. It can also have the ability to cure and prevent serious diseases that have plagued society for decades. Gene therapy has the potential to silence a gene before it becomes onset.

But there are also always disadvantages and risks when dealing will miraculous medicine. On top of that there are also always moral, ethical and legal lines that need to be put into place because of beliefs and humanity. One of the major turn offs is the fact that genetic testing is causing many ethical and legal problems amongst patients. For example, if parents do a genetic test on fetus and finds that it comes back positive for a terrible disorder than they could abort the baby because of it; which would then arise arguments of faith/beliefs, wellbeing, etc. There is also a money conflict, if a genetic test is positive, insurance can increase and costs can become unmanageable for the patient. There are also superficial consequences like if scientists are able to pinpoint phenotypic genes than gene therapy can turn into cosmetic medicine and overshadow the main goal to cure genetic diseases. On top of it all, gene therapy only lasts a short amount of time and needs to be administered multiple times to be as effective as possible and that can seriously take a toll on the human body and the immune system. Also, different people can have different reactions to the treatments like being immune to the vector being used or even allergic.

But overall a bumps along the way will pay off in the end when people with disorder will not only be able to cure themselves but the will also prevent the gene being passed down to any of their offspring.


Gene therapy is targeted to cure/prevent human genetic disorders and overall improve the quality of life. Because as mentioned before, gene therapy only lasts short amount of time and has to be administered multiple times to have the most effective results. These treatments can take a serious toll on the body causing exhaustion, major discomfort, pain and sluggishness.

Not only does it take a toll on the body but these treatments can take a toll on our environment. Gene therapy is also used on certain crops to ensure immunity and other advantages against the challenges in nature. These plants are called GMOs (genetically modified organisms) and an example of it is Round UP ready soybeans. Round UP ready soybeans are GMO soybeans that have been modified to be resistant to the weed pesticide Round UP. This can affect our bodies too because we could process the crop differently.

Economic effects of Gene Therapy are terribly consuming. There is a high as a million dollar price tag on on gene therapy and that can finically cripple some families. There is also the fact of raising rate in insurance and family situations like single mothers or fathers that can't afford expensive treatments like that.

The social aspect behind Gene therapy can be both good and bad. Because on one hand it is good and positive if disease are cured and population grows and reproductive age is reached. But on the other hand there is also the negative effects that the treatment has on the body which can still fail and further crippled the worlds population.