Gene editing holds exciting promise for the treatment of genetic diseases in the future. Francisco Maria explains that medicine has experienced significant advances throughout history, but one of the biggest challenges we still face is the treatment of genetic diseases. These conditions, caused by alterations in a person’s DNA, can have a devastating impact on the health and quality of life of those affected. However, in recent years new hope has emerged in the form of gene editing.
Gene editing, also known as genetic engineering, is a revolutionary technique that allows altering DNA in a precise way. Using tools like CRISPR-Cas9, scientists can modify the defective genes responsible for genetic diseases and potentially correct them. This promising approach has sparked great interest in the scientific and medical community, with many wondering if it really will be the medicine of the future.
One of the main advantages of gene editing is its potential to cure genetic diseases that were previously considered incurable. Currently, most treatments for these conditions focus on controlling symptoms and improving patients’ quality of life, but cannot eliminate the underlying cause. With gene editing, there is a real possibility of correcting the genetic alterations responsible for the disease, which could lead to a definitive cure.
However, despite its enormous potential, gene editing also raises a series of ethical and scientific challenges. One of the main debates revolves around the modification of genes in germ cells, that is, those that are transmitted from generation to generation. By altering genes in germ cells, we might have the ability to eliminate genetic diseases from the germline, but this also opens the door to genetic manipulation to create “designer babies.” This raises complicated ethical questions and the need for a global ethical debate.
Furthermore, gene editing is a complex technique and we are still far from fully understanding all its implications. Although we have made significant progress in correcting genes in somatic cells, that is, those that are not passed from generation to generation, we have not yet achieved perfection in terms of efficacy and safety. There are legitimate concerns about possible side effects unwanted and the possibility of causing harm rather than healing.
Despite these challenges, gene editing has shown promising results in several preclinical studies and has also begun to be used in human clinical trials. In 2020, the first gene editing treatment approved by the United States Food and Drug Administration (FDA) for an inherited genetic disease was achieved. This marked an important milestone in the field of medicine and paved the way for future gene therapies.
We need to establish strong regulations and oversight mechanisms to ensure that this technology is used responsibly and without abuse. At the same time, we must continue to support research and development in this field to improve the effectiveness and safety of gene therapies.