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Meta: Gene therapy could become the most important procedure known to humankind, check out our analysis of what gene therapy is and how it can help cure diseases.
Advances in medical technology have given us some of the most revolutionary cures for ailments that have plagued us for centuries. While not all serious diseases have known treatments, it’s through the persistence and continued advancement of our medical knowledge that allows us to get closer to finding those cures.
Gene therapy is an idea and practice that scientists believe will cure diseases that we still don’t fully understand and hereditary complications that we are born with. While it’s still controversial and is still seeking approval from the FDA, many doctors hope that it will become a practical approach to diseases and hereditary issues.
In this article will be discussing how technology is used to advance progress in gene therapy and what that means for the future of medical procedures. We will also take a look at how gene therapy is supposed to work, and the complications that doctors are facing that technology might be able to resolve.
What Is Gene Therapy?
Gene therapy is, in the simplest of terms, a process in which scientists take damaged cells and repair them to help fight off genetic disorders and serious diseases. The way that they manage to do this is by identifying the cells that are causing complications in our systems and then synthesizing a replacement copy of the faulty proteins to take their place.
Unfortunately due to how little we know concerning gene therapy and the process of replacing the damaged genetic code with good copies means that the risks associated with gene therapy are high. While in theory, gene therapy is a great way to treat horrendous conditions and diseases, and practice it can still cause more damage than good.
Some of the test results recognized some of the severe health risks that came from using the early stages of gene therapy. These results showed signs of the following:
For these reasons, gene therapy has remained a trial until there is a way found to stabilize the genes in the cell molecules.
There are ethical questions that should be answered as well before gene therapy can be considered a possible option for treatment. While the intentions for gene therapy are extremely noble, it wouldn’t be hard to use gene therapy for means that aren’t in any way considered a medical necessity and instead used as a vain attempt to enhance attributes of the human body.
How Does Gene Therapy Work?
Despite the ethical and complications that arise with gene therapy, the idea behind it is still quite a feat, and for the most part, scientists have figured out the gist of how it works. Taking a more in-depth look into how gene therapy works require a certain amount of understanding regarding how cell walls function.
For a mutated cell to be restored to working order, they must first extract the faulty protein that makes the cell mutated. Once the removal of a crude protein is complete, scientists have to find a way to successfully install the new copy of that protein to where it will function within the cell.
So far scientists have found two different ways to manage this by using a vector. A vector is a form of a genetically engineered carrier that allows the genes delivery to succeed and function within the cell. There are typically two ways to synthesize a vector, using a synthetic virus or a non-viral vector.
While this is a method that’s not as popular in use as viral vectors, however, some advantages can come from harnessing non-viral vectors to transfer proteins into the cell.
Large-scale production and the lower costs of the procedure create a compelling case for using this practice over the viral system, but for a long time, these processes weren’t able to compete with the success rate found when using viruses as vectors.
Recently, advances in vector technology have turned the tables for non-viral approaches to synthetic vectors and successfully improved the rate of successful transfer of copied proteins into cell walls. Currently, there are three main processes used to obtain successful non-viral vectors that will do the job. They include:
- Naked DNA
- Lipoplexes and Polyplexes
Using naked DNA is considered to be one of the simplest methods in non-viral transfections. While these trials were carried out with some success, the ratio of success to failures was lower than other ways that are mentioned in this article. Unfortunately, while the process seemed promising, the method’s success rate is too small to rely on entirely.
For those who are unfamiliar with the term, oligonucleotides are a polynucleotide which contains a small number of nucleotides in their molecules. They comprise roughly 20 of these in a nucleic-acid chain, and they’re used commonly in research involving genetic testing and forensics.
In gene therapy, they are used to inactivate genes that are involved in diseases creation and production. This practice has different ways to be achieved; one way is to use it to target specific genes that can disrupt the faulty gene.
They can also be used to signal cells to cut specific sequences made by the faulty gene so that they cannot communicate enough to affect the body. Scientists take this function further in their third method of applying oligonucleotides in effect gene therapy methods.
Scientists use a double-stranded oligodeoxynucleotide as a decoy to distract the transcription made by the mutated gene which reduces its ability to communicate with the rest of the body. Doing so prevents the ailment from continuing to produce problems for its host.
With lipoplexes and polyplexes, processes were developed to create a tumor suppressor gene that’s injected straight into the tumor. Studies have shown that this process has proven to be effective in decreasing the activity of things known as oncogenes which are the genes responsible for turning cells into tumors.
Usually, the most common approach to create a vector is from a virus that scientists modify so that the virus can’t infect the cells of the patient. Only certain types of infections qualify as a vector because they can enter the cells in question and successfully transfer the necessary protein into the cell.
Two prime examples of such viruses are known as a retrovirus and an adenovirus. Both of these viruses are used the most when it comes to these procedures.
Retroviruses can integrate genetic material from their cells into a chromosome that’s found in human cells, while adenoviruses can introduce their DNA into a cell’s nucleus but not into a chromosome.
These make ideal vectors because they have naturally occurring methods of entering a human cell which makes it easier for the corrected copy of the protein to work as intended. The vectors can be injected or given through IV into a specific area or tissue of the body where individual cells will take it up.
Scientists have also found that they can take a sample of their patients cells and expose them to the vector in a laboratory. Once the cells have the vectors, they then get returned to the patient where if the treatment proves to be successful, it will make a functioning protein.
How Does Technology Play A Part In Gene Therapy?
Currently, there are several different forms of technology in place that can significantly improve the chances of cells accepting properly formed copies of a protein that were deformed once before.
One such process is undergoing testing at Washington University in St. Louis where the researchers at the School of Engineering and Applied Sciences developed a method that allows more efficient insertion of macromolecules into the interior of the cell.
This technique is a blend of a process known as acoustic sheer partition or ASP for short and electrophoresis to make this happen. Utilizing this technique has shown over a 75 percent delivery efficiency of the copied protein into the cell which has been one of the most successful testings that have been achieved so far.
The reason that this works is that the ultrasonic vibrations that are made from the result of the two processes used in this treatment create pores that roughly measure 100 to 150 nanometers in size along the cell wall lining which allows the protein to enter the cell more easily.
In so doing, while there are pores created in the cell membrane, they’re not big enough to harm the actual sell itself while the delivery is accomplished. The same researchers were tasked with finding a way to use their research in therapies known as CAR-T cell therapy switch boost a patient’s immune system to ward off cancer cells.
While there are still many advances needed to push gene therapy into a workable solution for patients, the aid of technology is quickly bringing that dream into a reality for many people especially with the results that consistently prove that gene therapy practices are evolving to provide more successful results with every attempt.