What is it ?
Gene therapy is a method of treating genetic diseases or conditions. Genetic material, DNA or RNA, is used to help treat these conditions.
It can be implemented in two ways:
Altering the protein:
Gene therapy targets the root cause of the disease by giving the genetic material instructions on which protein to alter. This can adjust how much protein is made within the cells. They then use a vector (which is essentially a virus) to transport the new genetic material into the cell.
Killing of specific cells:
This can help treat diseases like cancer by killing the cells that are causing the condition. This is done by putting DNA into the infected cell so it will die.
The first way of doing this is by placing DNA that has the gene to make toxic products, so that the cell will be killed.
The second way this is done is by using DNA which can cause a protein to be expressed so that it can mark the cell causing the body’s immune system to attack the infected cell.
Gene therapy has been used to treat disorders such as hemophilia, cystic fibrosis, and sickle cell disease.
Issues of Gene Therapy
A major ethical issue in gene therapy is that it targets the embryo/germ line cells (germ line cells refer to the sperm or egg). This is a cause of concern because the process cannot be reversed. If regular cells are targeted this will only affect the person receiving the treatment and will not future generations. However, targeting embryo/germ line cells will affect future generations. This can cause other health problems or complications , and can not be undone.
The biggest concern for gene therapy is the expense. Currently, with the way medical procedures are priced, the worry that gene therapy will be unaffordable becomes apparent. Depending on how expensive it is these resources will most likely not be available to everyone. This puts those who aren't able to pay for gene therapy at a disadvantage.
Benefits of Gene Therapy
The main benefit of gene therapy is that various health conditions can be treated. This is especially beneficial if there is no other known solution (medicine, surgery, medical procedures etc.) to the condition. This is a long term cure since genes that are edited can not revert back to their original form.
Risks of Gene Therapy
Although gene therapy is helpful there are still risks involved. Some of these risks would need to be carefully considered before using gene therapy, since each individual's health is different.
Since gene therapy is usually done by using a virus to transfer genetic material, it can cause a negative immune reaction. This can happen since the person's immune system may see the new genetic material as an attack to the body. Depending on how severe it is, the body may start attacking itself.
Targeting of cells:
Targeting of cells is important and should be done with extreme care. The genes that are modified can not be undone, because of this the right cells need to be targeted to avoid any negative effects. If an already healthy cell is targeted it can cause complications that were not there in the first place.
1. A;, D. P. T. (n.d.). The economics of gene therapy and of Pharmacogenetics. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research. https://pubmed.ncbi.nlm.nih.gov/11873384/
6. What are the ethical issues surrounding gene therapy?: Medlineplus Genetics. MedlinePlus. https://medlineplus.gov/genetics/understanding/therapy/ethics/#:~:text=This%20approach%20is%20known%20as,that%20are%20not%20yet%20known.