Gene therapy is a medical procedure that replaces the genes that are infected on the host. Its main goal is to correct a disorder caused by a missing or abnormal gene. Despite it being in the experimental or early stage the results achieved have been quite encouraging. Gene Therapy Many genetic disorders and immune deficiencies are being treated successfully right now with gene therapy. 

The first gene therapy was approved by the National Health Institute in the late 1980s. Since then 3000 clinical trials involving gene therapy have been carried out. In 2015, 163 was under trial which has been counted as the highest number of trials done in a year.

95% of the trials are classified as being at an early phase of development. If targeted on the wrong cells its damage is much more severe. Everything comes at a cost, you have to pay whatever the outcome. Going on further detail here are some pros and cons of gene therapy.

Pros Of Genetic Therapy

1. Offers Hope for Infant

Genetic defects result in the infant death in the US, 1 in 5 are fatalities that’s about 3% of all births. It hereby clearly shows the diseases and disorders which are present in this current situation.

The birth defect which comes through genes is the cause of newborn death. Now as the gene therapy is getting to its high, it’s working towards reducing and correcting the defects and lower such heartbreaking incidents.

2. Treatment for Genetic Disorder

According to the stats, 10% of deaths in the total population is caused due to rare genetic diseases or disorders. There are more than 7000 distinct diseases and almost more than 78% of those are caused by faulty genes.

Gene therapy is capable of turning this ratio the other way, and eliminate the threat and provide an opportunity to prevent hereditary diseases like haemophilia and cystic fibrosis.

With this therapy, they replace the faulty cells with normal cells and provide appropriate treatment for the patient. Gene therapy opens the possibility for the solution to the problem which we thought were burdened by.

3. Treats more than diseases

It’s not only focused on solving genetic disorders or hereditary issues, but also provides the opportunity to the individual suffering from infertility to start its own biological family.

The modified gene therapy method called “CRISPR” carries the potential of altering infertility in humans one day. Identifying the defective cells in the patient and replacing those with the active and normal ones might help treat all the rare diseases. 

4. Diversified Options

Gene therapy research provides a solution for people who are suffering from specific illnesses and diseases. Once it starts solving these problems and gain successes in this field, a lot of other treatment areas might open up using this information.

The processes involved may help people struggling with vision or hearing issues. It can eventually relieve chronic pain, which would be tremendously beneficial for the future of many people.

5. New Potential Discoveries

There is much much more in the box to offer with the help of gene therapy as many people are involved in the research and study about its benefits. Gene therapy helps solve many issues and provides an opportunity for researchers, which leads to new ideas that might inspire critical discoveries and the results that might help future generations.

The diseases like Alzheimer’s, Aids, and Cancer are on the verge of being treated as the genetic therapeutic research is closing nearer towards the solution. Gene therapy offers the chance to treat numerous diseases and disorders that currently do not have a treatment protocol.

6. Breakthrough in medicine

In every minute and second the new unknown syndrome, disease or disorders are identified as the individual bodies react differently to the same treatment option.

Gene therapy offers the opportunities and opens the doorway for the treatment of specific diseases and disorders like haemophilia, leukaemia or severe immune deficiency issues.

Many chemical experiments have to be carried out to find the correct composition which might lead to the new medicinal breakthrough,

7. Not limited to humans

Gene therapy has expanded itself to the veterinary science field, it’s success has extended the lifespan of animals. It helps animals by providing protein when applied to livestock. This technology is used in plants so that they can fight against DNA and genes added to them.

It helps us to grow healthier foods, increase the life of harvests, and produce more items in our overall yields. Genetical treatment in these fields has helped animals and plants develop antibodies and fight for their survival and save from extinction.

8. Uses technology

Gene therapy has proved to be one of the best treatment options for doctors which is completely based on technology. Most of the medical discoveries rely on technological processes that are first tested on smaller animals. Now the advancement of genetic engineering or technology has developed new resources that can advance these medical treatments at a faster pace.

 As more research in this field occurs, prices will drop. This will benefit the lives of thousands of people and their quality of life. The spark of light and hope for the victim and its family will definitely change their mind about the problem they are facing.

Cons Of Gene Therapy

1. Complex

The virus vectors are used to deliver the gene in the host. Sometimes the transfer of genetic data can be eliminated by the immune system before it has the chance to work.

There is always a possibility for the patient’s body to react differently when introduced to a new substance but the risk is if it’s going to take it as an antigen or trigger a grievous immune response and attack back to the host.

The issues with cell division or replication might limit the effectiveness of the treatment.

2. Ethical Concern

Gene therapy leads to serious ethical questions as it could also be used to create “perfection” in children. If the parents have sufficient financial means they can genetically manipulate their children to be smarter or look a specific way.

Even if it is for the better, changing a person’s genetic profile eliminates the natural variations that occur within the generation. It might create a new social class of “created people”, which may bring a serious threat to a person or its generations to come.

3. Current gene therapy methods can sometimes target the wrong cells.

While gene therapy treatment virus is delivered to the human body which might affect more than one type of cell. If the healthy cells are damaged then the unpredictable result is to occur like disease development, illness, or even cancer. 

The first human gene transfer study was approved in 1989 by the National Institutes of Health. 800 gene therapy studies involved 5000 patients until 2006, the total number of success was zero.

They were successful to find a cure for “bubble boy” disease that also caused leukaemia by the virus that was transferred during treatment. 

4. Multigene Disorders

Gene therapy uses a virus vector to transfer genes into the body. They target the affected genes and replaces them with healthy ones but if the disease is caused due to mutation in multiple genes like in high blood pressure, diabetes, heart disease, and arthritis its difficult to perform gene therapy treatment.

If multiple genes are affected then it has less chance of success, as the targeted gene will only be cured and the rest would be infected which won’t help cure the disease. 

5. Humans could develop resistance to gene therapy.

The human body keeps on accepting and rejecting what it receives. It may grow resistance within itself, as happened with the antibiotics. When the antibiotics were first introduced it was very effective but as for now the approach for antibiotics has changed.

The patient would discover benefits while receiving gene therapy as the updated and healthy genes are transferred. It might be helpful for him only and not for the generations to come as the body would develop resistance to the information transferred.

6. Short Lifespan

While performing gene therapy the DNA is inserted into the cell and it starts working. But, the life span of the treatment is very low, once treated with gene therapy the patient has to be treated again after a certain period of time. Due to the rapid division of many cells, gene therapy faces the challenge of curing it for a long period of time.

7. Expensive

Like the equipment, manpower, chemicals, and infrastructure required for carrying these experiments and trials seems to be of serious concern which definitely shows how expensive it is.

Many therapies are stopped in between due to lack of funding, which delays the benefit that the patient was about to receive. These fees are not affordable to many, which might leave the people below the rich line with the burden of illness. 

8. No guarantee

Gene therapy is the most talked about topic in the recent world. It is just a concept that is in development or advancing stage gaining the attention of many people. As the numerous clinical studies and experiments are performed mostly on animals firsthand.

Now even if it is a success that’s not going to be the same for treating the human body how they treated the animals. So, there are many failures while doing this procedure on humans which shows that there is no guarantee of success.

Pros and Cons of Gene Therapy


(Last Updated On: September 5, 2020)
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