The Potential of Gene Therapy in Curing Disease



All living things degenerate in health as they use their molecular machinery to extract nutrients and energy from the environment. We can say that aging is an obligatory disease of living beings. There are many reasons for this natural aging, losing telomerase action encoded in our genome, being lost is the main one.


Telomeres are decreasing in size during cell division, so there comes a time when the genes begin to deteriorate. This is called Hayflick’s limit. Actually, any disease is a gradual breakdown of a living being's ability to maintain homeostasis, so that regaining the equilibrium of its internal environment becomes increasingly difficult until death puts an end to this eternal struggle between the living and the inert matter.


Of course, what we normally understand as disease are infections or congenital conditions that evidence an obvious lack of functionality. They differ from aging in that they can be fatal and abrupt, but at a conceptual level, it is the same inability to maintain that desired balance.


A method for maintaining homeostasis


If I told you that it is possible to reverse aging and any disease, infectious or not, what would you think? It is probably the most anticipated event for the lonely human existence. One cannot understand all the stories about the famous fountain of youth, or the elixir of eternal youth, without believing that the human longs for immortality.


Well, this capacity is a door that little by little is opening when we learn more details about the genome and its regulation. Keep in mind that since the first RNA molecule was manufactured, it has replicated without losing its homeostasis. All terrestrial living beings are descendants of these first news chains that managed to perpetuate themselves. The way he devised to get rid of the fatal entropy and be able to replicate his information is that all living beings die at a given moment, but also that they are born in a new generation. Therefore, we have that our genetic code can be preserved for millions of years with proper maintenance. Sure, this is easier in simple, single-celled organisms. Even in those cases, natural immortality is impossible as there are many factors that end the life organization. So, how do complex multicellular beings propose to cure diseases and reestablish this homeostasis?


If there is a formula, it must necessarily go through gene editing. In fact, there is open research on the subject of aging to add telomerase in aging individuals and reestablish the telomeres of their chromosomes, paralyzing this aging. It is not crazy to talk about this, although we are far from being able to heal and preserve all the molecular mechanisms of this vital "programmed obsolescence". There is still time to get there, but treatments for less complex and more abrupt diseases are now possible with gene therapies. I'm talking about diseases such as cancer, or without going any further: AstraZeneca's vaccines to generate immunity against SARS-CoV-2 are based on gene therapy with modified adenovirus, which contains information about a coronavirus viral protein.


What it is?


A gene therapy consists of any approach to cure a disease (not palliate it) whose basis is the introduction of gene material into the somatic cells of an individual. Typically, viral platforms or lipid vesicles are used to introduce gene material, although there are more ways to do this. The important thing is to understand the importance of designing well these sequences that we introduce. It must be an intelligent construct since it must avoid exonucleases as far as possible - enzymes that destroy gene information outside the eukaryotic nucleus - and integrate into the genome at a specific site, without affecting healthy functionalities. In the case of cancer, it can be a product that activates apoptosis mechanisms or that inhibits mitosis, for example. Congenital diseases can also be treated in this way, thus making it one of the most powerful and promising tools of medicine for the near future.


Current Status & Start-ups


Gene therapies are a definitive cure for previously untreatable diseases. Although it is still an experimental treatment, there are already many patients who have been cured with it. The plasticity and variability of execution in the technique allow adaptation to the unique needs of each person.


There are very interesting and promising Start-ups in this area, in addition to the public initiative. Generation Bio decides that the viruses normally used as information carriers are not large enough and limit the ability to modify the target cells, so they have invented another form called ceDNA that allows sending multigene information. Mediphage Bioceuticals, on the other hand, understand that gene therapies would require a specific supply of medication. They develop a safe and personalized way for you to redose your genetic treatment. Another different innovation is that of Cardior, as they seek treatments for other types of genetic diseases derived from the expression of non-coding RNA. These RNAs do not encode proteins and have regulatory functionalities. Therefore, its dysregulation is linked to the appearance of multiple serious diseases. Cardior uses adenovirus to deliver synthetic antisense RNA to put the genome back in order.


The Future


While genomics has come a long way, proteomics and transcriptomics have lagged somewhat behind. The future must contemplate the acquisition of new knowledge in these two fields of functional biology so that we can have a holistic approach, which necessarily goes through systems biology. It will not be immediate, but for each molecular problem, there must therefore be its solution. If we can solve every problem that deviates us from homeostatic balance, we will have found the longed-for elixir.


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