In this blog post, we will examine whether gene editing technology can realize humanity’s long-held dream of a future free of disease, as well as the possibilities and technical and ethical issues involved.
All humans dream of a future without disease. However, this dream has never been realized in human history. For many years, humans were powerless against disease, and “long life without illness” was only possible in myths. Even Qin Shi Huang, who ruled the entire world, passed away without finding the elixir of life, and even today, humans have not achieved complete victory in the fight against disease.
Although life expectancy is increasing, it is still clear that humans cannot completely overcome disease. Nevertheless, humanity has not stopped striving for a future free of disease. In modern society, a new “elixir of life” that will make this dream come true has appeared in the form of “science.”
Among them, medicine has studied the human body for a long time and proposed various treatments, but intractable diseases such as cancer and genetic diseases still remain. Furthermore, genetic diseases are passed down from parents to children and are considered to be problems that require prevention and fundamental solutions rather than treatment. In this situation, where the limitations of medicine are becoming clear, the possibility of overcoming disease is now expanding beyond the realm of medicine to the realm of engineering.
A technology that has been attracting attention recently is gene editing, which artificially alters the structure of genes. This technology is a new way to prevent or treat diseases by modifying human genetic information, offering an approach that is completely different from existing treatments. Gene editing has long remained theoretical, but over the past decade, it has undergone rapid development and is becoming increasingly applicable in practice.
Just a few decades ago, even deciphering the structure of human genes was a major goal of the scientific community, but now we have reached the point where we can select and modify or remove only the desired genes. At the center of this is CRISPR-Cas9 technology.
This technology was discovered in 2012 by researchers at the University of California, Berkeley, while studying the immune system of bacteria, and attracted considerable attention for its ability to precisely cut specific gene sequences. Since then, gene editing has become possible in cells of various species, and experiments have been successfully conducted in human cells, opening a new chapter in genetic engineering. In fact, in 2023, the US Food and Drug Administration (FDA) approved Casgevy, a CRISPR-based gene therapy. This therapy is used to treat genetic diseases such as sickle cell anemia and beta thalassemia, marking an important turning point in the entry of gene editing technology into actual medical practice.
This gene editing technology can approach diseases in two main ways. One is to edit genes at the fertilized egg stage to prevent congenital genetic diseases in advance, and the other is to treat diseases that have already occurred by modifying genes outside the body. Fertilized egg gene editing offers the possibility of fundamentally preventing the onset of diseases by removing genetic defects inherited from parents. Genetic diseases are often caused by mutations in genes, so if these can be corrected in advance, it may not be a dream to create a generation free of genetic diseases. However, as this technology deals with the earliest stage of life, the fertilized egg, it raises serious ethical controversies in that it permanently alters human genes. In 2018, Chinese scientist He Jiankui announced that he had created the world’s first gene-edited babies, causing a global uproar.
On the other hand, ex vivo gene therapy is a technology that is currently in practical use and carries relatively little ethical burden. This method involves extracting cells from a patient, editing their genes, and then reintroducing them into the body. A representative example is CAR-T cell therapy used to treat cancer, which manipulates immune cells to attack specific cancer cells. Recently, various ex vivo therapies utilizing CRISPR technology are being developed, and their application to diseases such as cystic fibrosis, Huntington’s disease, and hereditary blindness is becoming increasingly likely. In particular, this technology is considered a key to opening the era of precision medicine in terms of its ability to provide customized treatment for patients.
As such, gene editing technology offers new solutions in areas that were previously inaccessible to conventional medicine. Fundamental modification of genes goes beyond simply alleviating symptoms and acts to eliminate the causes of disease. Of course, this technology is not a panacea. It has not yet achieved perfect precision, and there are clear technical limitations, such as the “off-target effect,” in which unwanted areas are cut, and unexpected gene mutations. However, these limitations are gradually being overcome through technological advances. Recently, “base editing” and “prime editing” technologies, which are more precise than CRISPR, have emerged, increasing the accuracy and safety of editing. Combined with AI-based genome analysis technology, the practicality of gene editing is expected to be further enhanced in the future.
In addition to technical limitations, ethical controversies remain significant. In particular, concerns have been raised that gene editing could be used to manipulate essential human characteristics such as appearance, personality, and intelligence, beyond the treatment of diseases. The fear of so-called “designer babies” has given rise to distrust of science that is as great as the expectations for it. If we enter an era where humans can design themselves, questions remain unanswered as to what kind of humans we will create and what social repercussions this will have. However, as with all science and technology, ethical concerns raised in the early stages can eventually be resolved to some extent through social consensus and institutional regulation. Stem cell research was also embroiled in fierce ethical controversy in the past, but through technological advances, it has gradually gained social acceptance by shifting to the use of adult cells.
Ultimately, the direction and purpose of gene editing technology are at the heart of the issue. If this technology is used solely for the purpose of treating and preventing disease and is operated based on social control and ethical standards, it will contribute positively to human society. Technology is only a tool, and how it is used is entirely up to humans. Gene editing technology is now moving beyond mere possibility and is gradually establishing itself in the field of clinical practice and treatment. A future without disease is no longer a pipe dream. The most promising solution to the difficult problem of disease, which has plagued humanity for so long, has already been presented. That solution is gene editing technology. The task that remains is how responsibly we handle this technology. Gene editing technology, the most realistic and powerful scientific weapon for protecting human health and life, clearly shows the direction humanity must take in the future.
