12 Pros and Cons of Gene Therapy


Gene therapy is the process of transplanting genes that have developed normally in place of genes that may be missing or have developed abnormally to correct a genetic disorder. It is a technique that is still in its experimental stages, but has shown some promising results for some individuals. More than 2,300 clinical trials involving gene therapy have been conducted since 1989.

The advantage of gene therapy is that it provides an opportunity for affected individuals to lead a life that is “normal.” Some gene therapies may even offer the chance to survive because the genetic disorder is diagnosed as being terminal without an intervention.

The disadvantage of gene therapy is that, like any other medical procedure, it may not work. The experimental nature of these therapies means that the costs can be high to have them performed and many health insurance policies will not cover it because of the potential of failure.

Here are some additional pros and cons of gene therapy to think about.

What Are the Pros of Gene Therapy?

1. It offers hope.
Even with extensive screening in place for parents, there are numerous births that experience defects and genetic diseases. In the United States, 1 in every 33 babies experiences a birth defect of some sort. That’s about 3% of all births. Birth defects are the leading cause of infant death in the US, accounting for 1 in 5 fatalities. With gene therapy, some of these defects could be corrected to reduce these fatalities.

2. Genetic disorders can be treated.
Rare diseases affect about 10% of the general population. More than 30 million people in the United States are affected. There are over 7,000 distinct diseases that exist and about 80% of those diseases are caused by faulty genetics. With gene therapy, normal cells could replace the faulty cells and provide a legitimate treatment for the affected individual.

3. It may treat more than just disease.
Gene therapy techniques could provide individuals suffering from infertility with the opportunity to start their own biological families. Using a modified gene therapy method called “CRISPR,” successful alterations to fertility in mice have occurred. This creates the potential of creating a similar effect in humans one day.

4. It would create a new field of medicine.
Gene therapy offers the chance to treat numerous diseases and disorders that currently do not have a treatment protocol. Even age-related disorders that may have genetic foundations, such as Alzheimer’s disease, could be treated with the medicines and protocols that come from genetic therapeutic research.

5. Gene therapies aren’t limited to humans.
When gene therapies are applied to veterinary science, it becomes possible to extend the lifespan of animals. We could treat genetic conditions in animals to prevent loss. This would stabilize our animal protein food chain when applied to livestock. It could be applied to plants so that they can naturally withstand disease without additional DNA and genes added to them. The opportunities to help life are virtually endless, in whatever form it may take.

6. Gene therapy is based on technology.
Because gene therapies are technologically-based, their overall cost will drop as new methods and advancements enter the medical field. Initial treatments may be expensive, but the future of treatment in the coming generations may not be at all. As more research in this field occurs, prices will drop. We’ve already seen this with penicillin. When it was first introduced, the price was $20 per 100,000 units. With a typical dose being 4 million units, that’s the equivalent price of $70,000 per treatment. Today, the price of penicillin is just pennies per dose in many circumstances.

What Are the Cons of Gene Therapy?

1. It is a costly treatment option.
Even if gene therapy becomes an accepted form of treatment for the diseases that affect humanity, the cost of administering them could create socioeconomic classes that are based on wealth and health. Individuals could be tested at birth for genetic disorders and then classified into risk pools that may limit their access to care or provide them at an extreme cost. The first approved gene therapy treatment in Europe, Alipogene tiparvovec, is $1 million per treatment. Only one person had ever been treated with the drug since 2016.

2. Nature is adaptable.
As we have seen with the growing resistance to antibiotics, nature can readily adapt to changes that occur. Gene therapies may be useful now, but additional changes to genetic profiles could create unforeseen disorders in the future. There is no guarantee that the future potential of gene therapy can live up to its current potential to treat specific disorders. By manipulating genes, we could be creating new disorders for future generations without realizing it.

3. It may unlock unethical forms of science.
When humanity has the knowledge to manipulate a genetic profile, the science of eugenics becomes possible. It may create a future where children have their genetic profiles altered in vitro so that a specific result is created. This would create different “standards” in humanity, creating a different class of person who has been “perfected.” It would also highly the wealth gaps that exist in many societies because this service would only be available to those who could afford it.

4. Gene therapies have been stuck in trials for a generation for a good reason.
Many of the gene therapies that currently exist have been proven to be mostly ineffective. Conditions that are treated by a gene therapy improve for a short time, but then revert to the state they were before treatment began. For gene therapies that are successful, ongoing treatments or tissue donations, such as bone marrow, may be required.

5. It may encourage gene doping.
Although gene doping is not known to presently exist, it is a process that could equalize athletics or educational opportunities if equal access to the technology is given. If a person succeeds because of gene therapy when they may not have the same levels of success without it is a subject of ethical concern, especially when considering athletic competition.

6. It can provide a false hope.
Gene therapies have saved children from leukemia. In the case of Charlie Gard and others in a similar situation, the promise of gene therapy may provide a false hope of survival. There are times when saving someone may create an inferior quality of life. Is the person being saved for their benefit… or the benefit of their loved ones?

The pros and cons of gene therapy show us that there are some uncomfortable questions that need to be answered as this technology progresses. The hope it provides is extraordinary. There may also be unforeseen side effects that could cause more harm than good as we work to treat some of the most devastating conditions that humans experience right now.