What Is Gene Therapy And How Does It Work?

Thanks to modern science, there are many ways that we can modify our genes and try to tackle potential causes of hereditary diseases. Gene therapy is one of the ways that people can prevent harmful conditions from emerging as well as stop them from being passed onto future generations. 

There are a lot of reasons why you may want to consider gene therapy, and things that it can bring to your life. This is a brief guide into the process, and what it is typically used for. If you want to find out more about your genes, check out https://www.mybiosource.com/

What Is Gene Therapy?

Firstly, gene therapy is the process of treating sections of someone’s DNA strands that have been proven to cause diseases and hereditary conditions. The aim is to decrease the risk of passing something harmful and potentially life threatening onto later generations. 

A small amount of a particular disease’s genes are introduced into someone’s DNA to trick their body into creating the corresponding protein. This can challenge their previous genetics and increase the chances of receiving the therapeutic effect. 

The different types of gene therapy are classified by the two main types of cells. These are somatic and germline, somatic tends to be the most heavily researched form of gene therapy. This is because it affects all cells in the body apart from the reproductive ones, which means that the altered gene will not be passed onto future generations. 

Originally, gene therapy was believed to be more successful in infants, which is why treatment was typically delivered as a prenatal procedure. Treatment injected into the umbilical blood vessel, fetal tissue, or amniotic fluid. An ultrasound probe helps ensure accuracy and precision. 

Gene therapy is currently used for treating genetic diseases, and can follow the process of gene addition, correction, silencing, and reprogramming. 

Somatic Gene Therapy

This is the most common form of gene therapy, which is done inside the patient’s body. Because of this, it is known as in vivo. A particular cell’s pattern of gene expression is altered, and there are a huge number of scientific studies aimed at tackling immunodeficiencies, cystic fibrosis, hemophilia, and thalassemia. 

This is mainly because these genetic disorders are known as single gene disorders, which makes them slightly more straightforward to figure out a reliable treatment, or gene correction.  

Germline Gene Therapy

Another form of gene therapy focuses on reproductive cells such as sperm and egg. Functional genes are introduced into genomes, which makes future generations inherit the changes that are made. The focus is on correcting disease-causing gene variants before they can become problematic.

How It Works

There are a few ways that gene therapy is done, and there is still a great deal of clinical trials and research that is currently being done in terms of specific treatments. However, the approach typically involves replacing a ‘faulty’ or problematic gene which is known to cause a particular disease. 

This can be inactivated or replaced with a working gene. The functioning gene can be found through a donor, or within the patient’s own DNA from another area. 

Ethical Dilemmas

There are still a lot of concerns over the safety and effectiveness of certain gene therapies. This is because there are so many risks and potential mistakes that can happen during the process. For example, it is easy to target the wrong cells, introduce a virus into healthy areas, or damage other cells. This can lead to significant health conditions including cancer. 

Another huge area of ethical debate is germline gene therapy. It is illegal in many places around the world because the people who would benefit from the changes have not been born yet. Many scientists tread lightly around the subject because of this, and it continues to be a concern of people interested in gene therapy. 

The Future Of Gene Therapy

Currently, there is still a great deal of research that is going into the importance of gene therapy and the role it can play in healthy and unwell individuals. Diseases such as HIV are in trials and being tested, and there could be a new way of tackling life-threatening conditions before they have the chance to emerge. 


Gene therapy is an extremely complex topic because of the ethical concerns and risks that are linked with it. However, with more detailed research, it could be used to prevent the onset of many harmful genetic diseases in future generations.