What is Human Gene Therapy and
How Does It Work?
Genetic Medicine, Human Gene Therapy and
Treatment of DNA disorders
All of the latest findings and medical research in the field of genetics, DNA testing and molecular genetics. Plus discussions of the top medication and treatments.
In plain English, gene therapy uses the genes to prevent, treat or cure various diseases. As such, this kind of therapy is still treated as an experimental medical approach and technique. Gene therapy has attracted our attention for a reason. Imagine a day in not so distant future, where we will no longer use surgeries or drugs.
Instead, we will insert particular genes into our cells in order to cure a disease. It sounds like a scenario for a science fiction movie, but it’s not. Gene therapy is here. Here are some of the approaches, the doctors are already testing:
We put a lot of faith in gene therapy. We expect a lot. For instance, we hope to deal with the inherited disorders, cancer, viral infections, and similar challenging health conditions. We just can't help ourselves. Both the doctors and the general public are putting a lot of weight on gene therapy's shoulders.
It should be emphasized that for the time being, we are testing the gene therapy only for conditions, we don’t have other alternatives.
Gene therapy is also known as the therapeutic gene therapy. Or, we also refer to it as the genes application as a medicine. No matter how you approach or call it, you aren’t going to make a mistake. Gene therapy is all about the right use of genes. Its potential is simply overwhelming.
Gene therapy can easily turn out to be the next revolution in the medical science. There’s a chance that we can treat almost all kinds of genetic disorders.
The catch with the gene therapy is to identify, target, and treat the faulty genes that are responsible for a specific disease. You don't have to be a doctor to know that inheriting a mutated or let's call it a faulty gene is never the good news. At the certain point in your life, this faulty gene can trigger a serious disorder.
You got this one right. We're talking about cancer too. If we can replace these faulty genes with the healthy copies or new genes, then we can cure the condition. We have given you a clue to the next question you're about to ask.
How does it work?
Really, how gene therapies get down to work? All you have to do is to make a “switch” between the mutated and healthy genes. The truth is that this is something much easier said than done.
First, you need to identify the faulty genes. Then, you have to come up with an appropriate and healthy “compensation.” In simple words, you’re playing a dangerous game when you’re applying a gene therapy. Why? Because you’re dealing with the genes, that’s why.
When it comes to gene therapy, the things are far away from being simple. You should know that by inserting a gene into a cell doesn’t necessarily mean that the gene therapy will work by default. What you need is a carrier. What’s a carrier in gene therapy and why do we need it?
A carrier or a vector is a genetically designed component with a role of delivering the gene in order for it to work. Very often, the doctors are using the viruses as vectors. Of course, these viruses that work as carries are modified, so they don’t cause harm to the target cell. Instead, their “infection” actually becomes a way of delivering a healthy gene.
The vectors can be given intravenously or simply injected into a specific area of the body. There’s also one more way to do it. You can take out a patient's cell and “infect” it with a vector. Then, once you’re done with the combining of a cell and vector, you put it back all together.
If you have done the proper gene therapy work, you can expect that your new gene combination will make the proteins in a treated cell function the right way.
You have probably realized by now that there’s a long way ahead of the gene therapy doctors. There are so many technical questions and challenges to be solved in order for gene therapy to reach its full potential. For instance, we need to come up with new, better, and efficient ways of delivering the genes.
In addition, we need to precisely target the particular cells. Finally, we have to ensure that our body accepts and controls these new genes.
We can illustrate how does the gene therapy work with the help of following steps:
When you’re dealing with the genes, you need to be extremely careful. Our genes are the essential building bricks that provide invaluable information to our cells to produce enzymes and proteins, we need to grow and develop. The most important thing about our genes is that we need them to be and remain healthy.
When a part of a gene is missing or it is mutating, then your cells get the wrong information or no information at all what to do. As a result, you end up with a disease that can threaten your very life. Gene therapy is supposed to fix these mistakes caused by the faulty genes. Therefore, it’s not an exaggeration to say that the name of our future is gene therapy.