In A Nutshell: Gene Therapy For Infectious Diseases.

Imagine waking up to a radiant morning with severe symptoms that reveal to you that all is not well with your health. You manage to get yourself together, grab a cab and then head towards a nearby hospital. On seeing a doctor, he reveals to you after series of tests that you have a very health debilitating viral disease. With shock on hearing this and the thoughts hovering on your mind, the doctor tells you that there is simply no need to worry, but that does not even come close to calming you down. And then the doctor says, “what about gene therapy?” Unfortunately, you just look at him dumbfounded. 

So, what exactly is gene therapy?

In the following paragraphs, I will discuss the basic ideas about gene therapy, the promise it holds for a healthy future, its successes, limitations and gene therapy for infectious diseases such as COVID-19 which we’re currently facing. 

Genes make us who we are and they are composed of genetic materials such as DNA which is the building block that determines our physical expressions such as the color of our skins, height and even our immunity. Defects or shortcomings in our DNA could affect some of our physical activities and our immunity such as the way we walk or how we breathe.

 If DNAs are building blocks in the body, gene therapy helps in targeting the defects in these building blocks by repairing, inhibiting or replacing them with healthy ones.

Gene therapy for infectious diseases can therefore be defined as the introduction of healthy genetic materials, specifically DNA or RNA into our body to replace or inhibit the expression of the unhealthy ones that cause diseases when infectious agents replicate in the body.

Infectious diseases here are those directly caused by microorganisms themselves such as SARS-COV-2 which is a strain of corona virus that causes corona virus disease 2019 and those with bacterial or fungal origin. These are different from genetic diseases due to mutations or diseases due to toxins in the environment. 

It is very interesting to imagine the possibilities of having your DNA being tested for diseases and being able to eliminate such from your future. You may be curious about how genetic materials can be transferred into our cells during gene therapy. So let us dive into how it really works. 

Well, genetic materials cannot be directly transferred into cells but instead, they are introduced into a vector which is in form of a vehicle or shuttle that is used to deliver the genetic materials into the targeted cells to provide therapeutic effects, that is, to cure a disease or help your body fight a disease.

This can be done in two ways, either in-vivo or ex-vivo, using either viral or non-viral vectors which are the two major methods of shuttling the genetic materials into the cells. In the in-vivo method, the therapeutic gene-containing vector or vehicle is being directly injected into the patient. The gene is then expressed, producing a therapeutic protein for treatment. In contrast, the ex-vivo method involves the therapeutic gene-containing vector being introduced into the cell outside the body environment and then re-infused back into the patient. 

The use of viral vectors to transfer genetic materials has to do with using attenuated viruses, that is, viruses that have been stripped off of their disease causing abilities as vehicles while the non-viral vectors involve the use of other particles which are not viral but can be safely used to deliver genetic materials into the target cells’ DNA.

Gene therapy have been successful in treating a variety of diseases such as certain HIV cases, haemophilia, cancer etc. but while some breakthroughs have been seen, gene therapy also have some potential risks especially in its application for treating COVID-19. In the search for a COVID-19 vaccine, some of the pressing problems involved include how to use a viral vector in delivering the therapeutic genes and also, understanding how the immune system interact with the vaccine.

There could also be risks of the wrong cells being targeted and in the case of using a viral vector, the virus may regain its disease causing capabilities, among other possible side effects.

 

In conclusion, gene therapy as a field is still in its research arena with respect to various infectious diseases and still requires some research time to make sure that effective and patient friendly treatments are attained. With recent advancements in technology, some breakthroughs made so far and the number of candidates on the run to make gene therapy a success at improving patients’ health all over the world, this field holds great promise for a healthy future and can go on to liberate mankind from the shackles of various diseases.