The Future of Gene Therapy

It might seem like a stretch to think that we can access our genes to treat illnesses, but we’re entering an advanced age of medicine. Considering the timeline of genetics and our appreciation of it, it’s not so far off to think that we’re ready for a new breakthrough.

In 1859, Darwin published On the Origin of Species; one hundred years later, Watson and Crick uncovered the structure of DNA. We’re about 60 years ahead of that discovery, so who’s to say we aren’t approaching something new?

We’re going to look at gene therapy in this article. What is gene therapy going to do for us in the future? Where is it now, and why is it important?

We’ll explore those questions and more. Let’s get started.

What is Gene Therapy?

Let’s start by taking a look at the fundamentals of genes. Most of us know the general function of genes and what they sort of are, but the fine details are ones that we probably learned in middle school or high school.

If you were too busy thinking about sports, video games, and voice cracks to digest that information, don’t worry. So was everyone else. It’s foundational stuff, though, and it’s important.

Our genes are composed of our DNA. This is the genetic material that directs the body to do everything it does throughout the life cycle. From growing in our mother’s womb to being right-handed to breathing to digesting food, we’re compelled by genes.

We inherit two sets of genes, one from our mother and one from our father. There are up to 25,000 sets of genes in the human body, and the combination of genes that you have is specific to you. That’s what contributes to the vast amount of human diversity on planet earth.

Understanding Mutations

Genes have a knack for mutating. In fact, some of your 25,000 genes might be mutating right now as you read this. Those mutations can be good or bad. Plus, they can be inherited or passed down.

Mutations are what has driven human beings up to the tip-top of the animal kingdom, but they’re also what cause some chronic diseases, illnesses, and more.

When we’re handed those genes from our parents, or our own genes mutate to cause an illness, it’s tough to treat the source issue. It’s deep down in the building blocks of who you are, so it might seem like the issue is there to stay.

This is where gene therapy enters.

Gene therapy is the act of introducing, changing, or attempting to manipulate the genetic material of an individual. It’s not quite as dystopian as it might sound at first, though!

How It Works

To understand how to use gene therapy, we have to have a good understanding of the human genome. We also have to know the genetic structure of the individual and identify which genes are causing problems.

Further, you then have to identify or speculate on which gene it is that’s leading to the specific issue the individual is having. The job is to then replace that gene with an alternate one that will redirect the proteins of your cells are produced.

Remember that DNA just has the instructions for cells and groups of cells to do their work. Our bodies are comprised of cells, and each cell serves a particular purpose in the whole.

It’s possible to isolate cells or groups of cells and redirect them if you can identify their section of the individual’s genome. Once you’ve identified the problem gene, you can then replace it.

You can’t enter into the cell and replace the gene, though. That would require microscopic technology and an understanding of genes that we don’t quite have yet.

Instead, we have to use a vector to conduct the gene into the individual. Vectors are also called carriers, and they’re pieces of genetic material that can enter the body, bind to the cells, and adjust the DNA.

Understanding Vectors

A vector typically comes in the form of a virus. Viruses are excellent at entering an organism and lightly manipulating the genetic material.

The virus, in this case, has been modified by scientists to eliminate the parts that make it spread to others or make a person sick. Instead, the target gene is introduced to the virus, the virus is introduced to the individual, and the individual receives the target gene.

Biologists and professionals can use RNA-seq data to identify how and where to place genes, remove harmful ones, and much more. Once they do that, they have an actionable way to fight difficult diseases.

That’s all.

In Vivo vs. Ex Vivo Therapies

There are two kinds of gene therapies, though. In vivo therapy is the sort that we described above. The cells remain in the patient’s body while the gene is introduced through a vector.

In ex vivo therapy, the cells are removed from the patient’s body; the target gene is introduced, then the cells are put back into the body. The version used on a particular patient depends upon what issue that person is experiencing.

Some diseases and ailments might be better treated with ex vivo therapy, while others might call for a less invasive approach.

Current Applications of Gene Therapy

Now, it might seem as though gene therapy is a cutting-edge idea or that it’s not commonly used on patients.

In fact, there are several diseases and illnesses that are treated with gene therapy and have been for a while. For whatever reason, active gene therapy examples aren’t discussed as much you might think they would be.

Issues like HIV, cancer, GBN, immune deficiencies, and more are actively treated with gene therapy at this moment. Millions and millions of people have received gene therapy thus far, and that number continues to grow every day.

This is because gene therapy works! What better way could there be to treat issues that stem from the genetic material of an individual? Seeing as genes are what direct every function in the body, it makes sense that we would use them to direct the treatment of fundamental issues.

We might not talk about gene therapy as much because it doesn’t quite look how it sounds. We think “gene therapy,” and we imagine doctors changing fundamental pieces of our biology, looking at particular DNA sequences and manipulating them to create new individuals.

Instead, the reality is just a very practical way to address health issues. When we look to the future, though, we might start to see some of those more fantastical things.

What Does The Future Look Like?

We can think of the future of gene therapy as we think of a lot of different things.

A good analogy to use is that of a house. A house is built with thousands of different pieces, materials, and arrangements. There’s a foundation made of concrete, a structure made of wood, interior walls, insulation, lights, switches, carpets, furnishings, appliances, and a whole lot of nails.

Similarly, a city comprises people, various communities, establishments, roads, sewers, power sources, and more. Our bodies are composed the same way.

In the case of cities and houses, we can look at particular problems and address them as we see fit. A light goes out; you replace it. There’s an issue with inequality in the city, and we can diagnose ways to change how the establishment treats people.

We do this with our bodies to an extent, but a lot of the issues we experience aren’t treated in simple ways. While we’ve made a lot of progress in medicine, there is still a slew of conditions that aren’t treatable one hundred percent of the time.

The future of gene therapy might allow us to direct treatment with a lot more efficacy.

Examples of Future Treatments

While we can’t know exactly what’s to come, we can imagine a rough sketch of how gene therapy will look in the coming decades.

For one, we can expect real advancement in the treatment of genetic diseases. Anything that’s inherited through genetic material will be better understood and more treatable.

Issues like muscular dystrophy, Alzheimer’s disease, sickle-cell anemia, and melanoma might be curable. Further, those issues will be evident sooner in the person’s life. A deeper understanding of an individual’s genome will allow doctors to take preventative measures and ward off the issues that come later in life.

These issues might even be visible when the person is just a child. A look at the genetic material of an individual will tell doctors what sort of gene therapy to give early in life, preventing painful and deadly conditions later.

As time goes on, those treatments will get more accurate and simple to conduct. They might also become very affordable things to have done.

Increased Specificity

Beyond the treatment of major illnesses, gene therapy might become more specialized and applicable to small situations.

Most illnesses are localized to a part of the body, no matter how small. That part of the body is composed of cells that are directed by genetic material. It follows, then, that an advanced understanding of gene therapy could allow doctors to treat smaller things with gene therapy as well.

Let’s say you have a bad rash. There may be a way to use gene therapy to boost those cells’ ability to repair themselves and eliminate the rash quickly. You might be able to cure the flu just a few minutes after you start experiencing symptoms.

Your foot keeps cramping up at soccer practice, so you have gene therapy to address those cells and muscles. The potential is almost endless.

In a hundred years or so, we might find that our way of practicing medicine is very dated and archaic in comparison to the methods that gene therapy gives way to.

Ethical Implications of Gene Therapy

We’re not yet in the territory that philosophers might debate over when it comes to manipulating the genes of human beings.

That said, the time when we’re choosing to alter people’s genes for reasons other than medicine could be coming up. A tweak here, a gene there, and you might be able to create a bunch of people who are taller, stronger, smarter, and more capable than others.

You might be able to get an alteration that makes you more alert and productive at work. Maybe you want to be more attractive to potential partners.

In any case, taking those liberties in a society will lead to some sort of inequality. It may also be the case that only the privileged are able to use gene therapy to change themselves, exacerbating differences and stratifying things even more.

Gene therapy is a purely beneficial thing when used to treat suffering patients. That said, the futuristic potential that it has might be alarming to some people. We probably won’t experience those things in our lifetimes, but there’s a world where we could pull at the strings of nature in those ways.

The trouble is that we could pull on those strings so much that they snap. What if we could engineer clones that were stronger, smarter, but more subservient? What about people who were modified to love doing manual labor?

Books like Brave New World detail these dilemmas and paint a picture of what they might look like. Of course, creating versions of human beings to do your bidding has profound ethical problems, but we experience serious problems already and choose not to do much about them.

Fortunately, we’re a long way from that point. The future of gene therapy for medicinal purposes, though, should give way to a healthier human race.

Want to Learn More?

Gene therapy is a complex thing, but it’s exciting to learn about the cutting edge of medicine. We’re here to help you learn more about gene therapy cancer treatments, gene therapy companies, and more.

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