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Gene Therapy Simplified: What It Is and How It Is Applied?

Suppose you have been keeping up with the various scientific communities and the many developments in the area explicitly concerning biotechnology. In that case, you must have come across some of the buzzwords that include ‘Gene Therapy,’ ‘Agricultural Biotechnology,’ and more.  We are living in exciting yet dangerous times. Exciting because of the technological and […]

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Suppose you have been keeping up with the various scientific communities and the many developments in the area explicitly concerning biotechnology. In that case, you must have come across some of the buzzwords that include ‘Gene Therapy,’ ‘Agricultural Biotechnology,’ and more. 

We are living in exciting yet dangerous times. Exciting because of the technological and biological advancements is paving ways towards a secure future for living organisms in general; Dangerous because of the biological threats that are proving to be deadlier than a conventional war, including COVID-19 has progressed into more destructive variants among global warming. 

Biotechnology, in specific, has been making considerable contributions towards medical sciences, be it enhancing immunity against serious diseases, providing genetically improved treatments, or working towards producing genetically modified crops (GMO crops.) Just like these, one of the fascinating progress that this domain is working on is Gene Therapy

Before we get into what gene therapy is, let’s briefly discuss what exactly is a gene?

What is a Gene?

In school, we teach kids that genes carry information that determines their traits, including features and characteristics passed on to you from your parents. Taking this definition a bit further, it can define genes as the genetic code passed down or inherited from each of your parents. The nucleus in the cell contains this genetic code. 

Genes contain instructions for the cell on how to make the necessary proteins. These proteins are needed to perform most of the life functions that include our development process, controlling our growth, and determining how we will look along with instructions on body changes till death. Not only this, but they are also responsible for repairing the damaged cells and tissues. 

However, when the gene itself is mutated or damaged, it is known as a mutation. A mutated gene then causes problems if growing uncontrollably, developing cancer. So, how does gene therapy is planning to right such wrongs?  

What is Gene Therapy?

Gene therapy is an experimental technique that includes the replacement of abnormal genes with healthy counterparts, which means that if a gene has mutated or gone rouge or missing, gene therapy may introduce a normal copy of the gene to restore the balance and functionality of the protein.

If successful, the doctors will treat patients with gene therapy instead of relying on drugs and surgery. 

There are several approaches under experimentation for gene therapy, including: 

  • Replacement of mutated or corrupt gene with a healthy gene. 
  • Inactivating a mutated gene that is malfunctioning; deleting it entirely. 
  • Introducing or injecting a new gene into the body helps fight diseases or protect from the harmful side effects of therapy like chemo and radiation. 
  • Creating ‘suicide genes’ that can enter the cancer cells and self-destruct those mutated genes.

Imagine if this therapy becomes a success and a possibility. The number of diseases that are still lingering on for a long time, like Alzheimer’s, cancers, diabetes, AIDS, and many more, will be treatable, halting the ‘genetic inheritance’ of such deadly diseases and disorders in the future generations to come. 

Currently, gene therapy is being experimented on diseases that have no cure at all. One of the matters of concern that remains largely is the uncertainties that come with changing the gene structure. The clouded side-effects could be one too many that can or cannot be worrisome. Nonetheless, this is why the research is underway, along with heavy experimentation. 

Now that we know what gene therapy is, we should speak about how exactly it is carried out. Interesting, isn’t it? 

How Gene Therapy Works?

Unlike its definition, Gene therapy isn’t as simple as picking a bad gene and then replacing it with a healthier one. It does sound easy on paper, but gene delivery, in reality, is one of the biggest challenges of gene therapy. Considering how small the cells are, it is hard to inject genes directly into the tiny cells. This is why a carrier or a ‘viral vector’ has to be altered to carry human DNA. 

This new genetic or working gene is delivered into the cells by using this viral vector. 

What is a viral vector? 

It is a tool that is used to deliver genes into the cells. This tool is a non-contagious virus. Since viruses attack their host and introduce their genetic material into the host cell, this newly injected genetic material has instructions on producing copies and hijacking the body’s average production. The host cell will carry out these instructions and start creating copies of the virus infecting more cells. 

Considering this pattern where the virus naturally infects the host cell with their genetic material, it can correct genes as well. 

First, a scientist would remove the pathogen genes present in the virus that are responsible for the disease and replace them with the desired gene pattern or encoding. This entire process needs to be handled quite carefully. 

Risk Factors involved in Gene Therapy

The above process was oversimplified for a layman’s understanding. However, gene therapy, in its essence, is complicated and poses many problems and risks. For instance, viruses can usually infect more than one kind of cell. This uncertainty can lead to viral vectors infecting already healthy and mutated genes with the new genetic coding they were injected with. 

Additionally, another significant threat that looms is these viral vectors being inserted in the wrong location of the DNA that could cause more unknown mutations and give rise to deadly diseases leading to death in some rare cases. 

AUTHORBIO

William Patrick Slattery, President and CEO at NIEUW AMSTERDAM ADVISORS

William Slattery brings over 35 plus years of senior executive experience to his position as President and CEO of Nieuw Amsterdam Advisors. He is regarded as one of the top marketing experts in the Life Sciences industry noted for combining a calm demeanor with a shrewd negotiation skill set that allows for navigating the most challenging business environments on behalf of his client firms.

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