The term “genetic modification” has been the subject of controversial and controversial scientific, religious, political and cultural debates. However, the techniques that are currently being tested in laboratories and clinical trials around this technology seem to be materializing what humanity wants so much: the cure of inherited diseases and the delay of the effects of aging.
Geneticists are very optimistic about turning to genetic modification to treat Alzheimer’s or Huntington’s disease, as well as to deal with cancer. In fact, they say that gene therapy will very soon be a viable option for those who suffer from a genetic disease and want to be cured.
In this way, it is designed that when these techniques are approved and their use guarantees the safety of the human species, some of the advantages that can be obtained will be the realization of faster and more precise diagnoses, personalized treatments, the prevention of diseases caused by the mutation of a gene and even the improvement of the capacities of the human being.
Now, what techniques are the most effective to carry out genetic modification? Currently the so-called CRISPR-CAS 9 is in charge. It is a method discovered by Jennifer Doudna and Emmanuelle Charpentier (Nobel Prize winner in Chemistry in 2020) that consists of eliminating or replacing undesirable genomic parts in order to edit the genome pursuing a therapeutic and / or medical objective. As such, “CRISPR” is the name of repetitive sequences present in the DNA of bacteria, which act as autovaccines. They record the genetic material of the viruses that have previously attacked the bacteria and in this way they can recognize it in the future to attack them by cutting the DNA of the invaders. The breakthrough is that geneticists have learned to use such molecular scissors outside of bacteria to cut and paste pieces of genetic material into any cell.
According to Lluís Montoliu, researcher at the National Center for Biotechnology of the CSIC in Spain, one of the exponential advantages of this technique, compared to the old ones that were very laborious, unpredictable and expensive, is that it “is simple and cheap, and it is not they need special equipment to apply it ”.
However, scientists around the world agree that its use must be regulated in view of the fact that although the outlook is very encouraging and promising, the nonspecific corrections that the therapy can cause must be controlled.
One of the examples of treatable pathologies that is in vogue is that of transthyretin amyloidosis: a multisystemic disease caused by mutations in the transtiterin gene (TTR), a characteristic that makes it a perfect model to be studied because it is of genetic origin and it depends on a single gene.
Another benefit of this clinical study that is in the first phase is the mode of administration of the treatment since the researchers assure that it is possible to apply the CRISPR / C9 technology to the patient through an injection into the bloodstream without affecting or performing changes in other parts of the body or genome. So far the responses given by the six patients in the experimental phase of the therapy have been very encouraging. However, there is still a long way to go and analysis to make references to long-term efficacy and possible side effects, such as unwanted mutations in DNA sequences or in other tissues that could affect the offspring of the recipient of the therapy. . This consequence is unlikely as error rates have been shown to be very low.
On the other hand, more than forty clinical trials have been registered around the world to treat about 50 diseases, such as HIV, congenital immunodeficiencies such as chronic gramulomatosis, leukodystrophy, sickle cell anemia, cancer, tuberculosis, Hereditary blindness and Kabuki syndrome.
However, a milestone that raised the global alarm in 2018 was the announcement of the Chinese biologist He Jiankui who claimed to have genetically modified embryos to obtain the first gene-edited human babies (twins) with a presumed capacity for resistance against HIV.
From then on, various entities spoke out and demanded the creation of an international framework to regulate genetic editing without disrespecting the rights of both individuals and present future generations wherever they may be.
Finally, after two years, the World Health Organization (WHO) released the first global recommendations for gene editing in humans as a public health tool. To make it possible to stipulate these resolutions, scientists, patient associations, and spiritual leaders met in order to establish the regulatory and ethical aspects related to genetic modification and its impact on research and biomedical practices.
Among the suggestions of the new document, it stands out that the clinical trials of genome editing be reviewed and approved by research ethics committees and that the manipulation of the hereditary genome is only carried out in places governed by these supervisory entities.
Scientists in laboratories are constantly advancing and that is why it is not surprising that there are already so-called CART therapies, which consist of safely and precisely applying drugs resulting from genetic manipulation to prevent the proliferation of cancer cells in, for example , patients with leukemia.
Following the order of scientific advances, it should be noted that Harvard University researchers developed a new gene editing technique called ‘prime editing’ that allows modifying the genetic code of a cell to eliminate both possible failures in its functioning as the diseases that it may cause. In fact, it is predicted that this method that avoids possible mutations could replace the aforementioned CRISPR used since 2012.
To conclude, it is important to emphasize the need to gradually form and establish multidisciplinary work teams made up of health professionals, geneticists, various clinicians and bioinformaticians capable of offering this type of medicine and therapies in environments supervised and regulated by policies that guarantee the safety of all, including future generations. For its part, IC Corporation, always at the forefront, supposes not only a human, economic, and technological effort, but also a specific style of conceiving business activity, and ours aims to contribute to the progress of societies and the development of people. In the countries where we work through its philanthropic arm, the IC Cares Foundation, it will continue to be responsible for supporting research and scientific development with resources in order to achieve solutions, cures and effective treatments for the various diseases that afflict the population. .
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