This is a question, we just couldn’t avoid asking. We have high hopes when it comes to gene therapy and a realistic possibility to treat serious and life-threatening diseases. However, we need to make sure it is totally safe with no side-effects whatsoever. When you’re dealing with the genes and cellular level you’re playing a dangerous game that can save someone’s life, but also open a Pandora’s box. The goal is simple. Gene therapy has to be safe and efficient.

The ultimate prize is just too tempting to ignore. Can you imagine the world with no cancer, Parkinson’s, Alzheimer’s or muscular dystrophy?

Well, we’re pretty much convinced that this is the main motivation for thousands of scientists who are working around the clock in this field with this particular vision in their minds.

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New revolutionary approaches are always risky

Gene therapy isn’t a new concept from the theoretical aspect of view. For quite some time the scientists have examined the possibility to treat the diseases with the gene therapy.

Nevertheless, the first practical applications have taken place over the course of the last few decades. There’s still so much work to be done. The outcomes are still unpredictable. We can’t say for sure how gene therapy will work.

Right now, we are careful in applying gene therapy results. What does it mean? The FDA (the US Food and Drug Administration) didn’t want to stay behind. That’s why, the FDA strictly regulates the products and solutions obtained as a result of gene therapy applications.

In addition, if you are a US scientist and you want to conduct some clinical trials in this field, you have to ask the FDA’s approval first. It sounds reasonable that the FDA’s authority in this field is unquestionable. This agency can suspend or reject any gene therapy clinical trials that are considered to be potentially harmful.

The FDA is not the only agency that's paying a close attention to what's happening in this field. We should also mention the NIH (National Institute of Health). This agency has issued a series of guidelines and instructions that the gene therapy researchers have to follow and comply with.

Additionally, all studies that are funded by the NIH have to be registered with the Office of Biotechnology Activities at the NIH. So, are we safe then with so many agencies and procedures that are watching over us?

It’s risky business

It’s risky business, but it’s worth all the trouble

In its essence, gene therapy is actually quite a simple procedure. You find the faulty genes and you replace them with the healthy alternative. That’s the theory. The practical part is a bit more tricky. First, you need to precisely locate and identify the faulty genes that are causing the disease in the first place.

Then, you have to find a way to deliver a healthy alternative. We are talking about a “vehicle” or vector. That’s usually a virus that is delivering the healthy genes to the target cells. Finally, you need to make sure that these healthy genes do their job right.

Now, you see that gene therapy is a complex and risky process. There are so many phases of the process where something can go wrong. For instance, you can identify the wrong group of faulty genes. Or, what’s even worse, you carrier a virus can cause more harm than good.

That’s why the so-called vectors in gene therapy have to be modified that they are no longer viruses in their basic form. All of their good penetration characteristics are preserved and not one of their harmful components is left behind. That’s the plan.

Beware of the potential side-effects, but don’t forget the ultimate prize

Nurse checking man's temperature

In the worst case scenario, your body isn’t going to be thrilled with the new “guests” who are paying a visit with no invitation. Our organism may fight the new genes. What’s even worse we may develop a reaction to a virus, which is a carrier of the healthy genes. The next thing you know, instead of a gene therapy cure, you may end up with an additional health problem.

The problem is that we have so much more to learn about our genes. You can successfully apply one gene therapy on hundreds or even thousands of patients. However, it only takes one patient, who takes it badly, to compromise your entire gene therapy approach. In that case, you are back where you started.

It's just like you are walking through a huge minefield and you are leaving the flags behind you where's safe to pass. The trouble is that you have to do it step-by-step and the clock is ticking. The patients and the general public are impatient for a reason. Yet, there's not much we can do about it. We just have to keep trying and experimenting.