American scientists successfully transform human embryos
Release date: 2017-08-04
Today, Nature published a highly topical study online. A multinational team from the United States and South Korea used the popular CRISPR-Cas9 gene editing system to repair a genetic mutation that causes heart problems in human early embryos. This means that we finally have a safe and reliable means to completely change a person's genes, and also let us be close to the role of the "creator." As many have been eagerly awaiting, this study touches the boundaries in both science and ethics.
Professor Shoukhrat Mitalipov, one of the main leaders of the study (Source: Nature)
Epoch-breaking breakthrough
In this paper in Nature, researchers want to repair mutations in the MYBPC3 gene in human embryos using the CRISPR-Cas9 gene editing technology. The probability of this genetic mutation is not low - according to statistics, one out of every 500 people is affected by it. It can cause hypertrophic cardiomyopathy, causing a seemingly healthy young man to suddenly die.
Since this mutation is dominant, it can easily pass to the next generation and cause the same symptoms. Therefore, if we can use genetic editing to repair this gene in the early stages of human embryonic development, we can eliminate the hidden dangers caused by this mutation once and for all.
Researchers want to use the CRISPR-Cas9 technology to repair a patient's genetic mutation (Source: Nature)
To achieve this idea, the researchers first developed the CRISPR-Cas9 gene editing technology for hypertrophic cardiomyopathy. They recruited a male patient and then attempted to remove the MYBPC3 gene mutation in his skin fibroblasts. The development of the technology is very smooth, and the repair work can be completed in two steps: first, the mutated gene region is cleaved by the Cas9 enzyme; secondly, using the artificially provided normal MYBPC3 gene as a template, the intracellular DNA repair mechanism will The mutation is automatically repaired.
Later, they tested whether the technology could work in human embryos. The researchers combined the male patient's sperm with the donated egg to fertilize the egg. After 18 hours of fertilization, they injected a set of systems that could repair the MYBPC3 gene into the fertilized egg. Subsequently, they evaluated whether the gene was effectively repaired in multiple embryos at a single cell resolution.
The design idea of ​​this research (Source: Nature)
The results of the experiment are shocking! The system repaired genetic mutations in embryonic cells at a very high rate, and the researchers did not detect significant off-target mutations or genomic instability. This shows that this multinational team has developed an effective technology for transforming human embryos without creating common problems that people have previously worried about.
Professor Juan Carlos Izpisua Belmonte (left) from the Salk Institute is one of the principals of this study; Dr. Jun Wu (right) is the co-first author (Source: Salk Institute)
"Because of breakthroughs in stem cell technology and gene editing technology, we have finally begun to solve some of the genetic mutations that cause disease. This is expected to affect millions of people around the world!" One of the co-authors of the paper, Juan Carlos of the Salk Institute Professor Izpisua Belmonte said: “Gene editing technology is just getting started. Even so, we have confirmed its safety and effectiveness in the preliminary results. The key to the future is that we must concentrate all our attention with the utmost care, To consider all ethical issues."
Uneasy and worried
Any breakthrough technology will cause heated discussion when it comes out, let alone this technology to directly transform human embryos? For this research and its future development, people's concerns are mainly in two aspects.
The first is in the gene editing technology itself. At present, we are still unable to fully understand the response of embryonic cells to the CRISPR-Cas9 gene editing technology. For example, in this experiment, the researchers provided a man-made DNA with a healthy MYBPC3 gene. It is envisaged that this DNA should serve as a template for genetic mutation repair. However, what they expected was that the cells did not adopt this artificial template, but repaired the MYBPC3 gene mutation based on the healthy genes in the egg. In other words, although the experiment yielded satisfactory results, we did not control every aspect of it.
Prof. Feng, a pioneer of the CRISPR pioneer, calls for human genome editors to be cautious (Source: Stat)
In addition, the individualization of gene editing is also the focus of many scholars. A few days before the publication of this paper, Prof. Feng, a pioneer of CRISPR technology, wrote an article in Nature Medicine, calling for caution when editing human genomes. (Related reading: Prof. Zhang Feng’s issue of Nature’s publication : Editing the human genome requires caution). Because of the large number of genomes between people, genetic editing specific to one person may cause off-target effects in another. Therefore, Professor Zhang Feng suggested that genome-wide sequencing should be performed before gene editing to maximize the specificity of editing techniques. In the human embryonic stage, whether such a strategy is feasible or not requires further verification.
“To be honest, I feel a little uneasy,†commented Professor Jennifer Doudna, another pioneer of CRISPR technology. “This is not about research. I mean how we apply this technology to the clinic.â€
Professor Jennifer Doudna, another pioneer of CRISPR, expressed concern (Source: University of California, Berkeley)
Other scholars have raised considerations from an ethical perspective. They pointed out that we must use CRISPR technology to achieve our goals? Since the MYBPC3 gene mutation is dominant, for the carrier of the heterozygous mutation, the probability of inheriting this mutation to the next generation is only 50%. Since in vitro fertilization techniques are also used, we can completely screen out embryos without this mutation and eliminate the hidden dangers of the disease.
In addition, some scientists worry that we are going too far on the road to transforming human embryos. In this study, we just used genetic editing techniques to fix genetic mutations in the disease. In the future, what happens if someone wants to use this technology for other purposes, such as improving people's intelligence or enhancing their physique? "Artificially designed" babies are bound to cause ethical and intense discussions.
postscript
In any case, the research itself has important scientific value and significance, and it is enough to become one of the annual scientific breakthroughs. Many scholars, including the authors, suggest that we need to know more about the mechanics and risks of this technology. Even if it goes into clinical applications in the future, we still need to strictly supervise it.
Whether the study brought a gift from the goddess of science, or opened the Pandora's Box, the future will give us the answer.
Source: Academic Jingwei
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