Gene editing and cancer treatment enter clinical trial stage August 31, 2016 Source: Xinhua Daily Telegraph Zhoushan Fudan Tourism CO., LTD , https://www.fudanfood.com
Gene editing and cancer treatment enter clinical trial stage
Gene editing "can exchange species DNA for what we want", relying on it, humans can not only achieve rapid directed breeding of animals and plants, but also promote the subversive revolution of disease treatment, and obtain the "writing of the life script". In the near future, human beings are expected to turn incurable diseases into chronic diseases that can be controlled for a long time.
Not long ago, the Lu uran team of the Huaxi Hospital of Sichuan University will conduct the world's first human-based CRISPR gene editing clinical trial news, making gene editing technology once again the focus of public attention. What is genetic editing? What does genetic editing bring to humans? What is the status of China's gene editing technology? The reporter recently interviewed industry experts and tried to uncover the mystery of genetic editing.
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Magical "scissors" will rewrite life scripts
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"Gene editing technology is a 'scissors' for human cutting genes." Song Yangzhou, a professor of the first batch of "Thousand Talents Program" of the Central Organization Department and a professor at the School of Life Sciences of Sun Yat-sen University, said.
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Gene editing technology began with the gene targeting technology established in the 1980s. After 2000, ZFN and TALEN technologies appeared successively, which can achieve high-efficiency directional clipping on the genome. After 2012, the third generation gene editing technology CRISPR-Cas9 Appeared, this new technology has high precision, low cost, and simple operation, which has greatly reduced the threshold of technology. The world has thus set off a research and experimentation on genetic editing.
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According to reports, new gene editing technologies, including CRISPR/Cas9, can quickly, efficiently trim, cut, replace or add DNA sequences. Because the gene editing "can replace the DNA of the species with what we want", human beings can not only achieve rapid and targeted breeding of animals and plants, but also promote the subversive revolution of disease treatment and obtain the "writing of the life script".
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Ma Wenbin, a professor at the School of Life Sciences at Sun Yat-Sen University, told reporters that the species on the planet use a highly similar genetic program system, and genetic editing technology has broad application prospects. With nearly 30,000 protein-coding genes in humans, scientists can obtain a "map" of human genome function through knockout experiments in model animals.
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Dr. Han Xu, from the Human Gene Gene Editing Department, said that through the Human Genome Project, people read the "life code" and learned the meaning of some DNA codes. After "can' read", the researchers wondered if they could change the code, such as making wheat resistant, such as eliminating human diseases."
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In recent years, despite ethical controversy, countries around the world have rushed to break through the original rules and regulations and actively promote genetic editing research. In April 2015, Huang Jun, a professor at the School of Life Sciences at Sun Yat-Sen University, completed the world's first genetic modification of human pathogenic genes in the world, which triggered the attention of the global scientific community. Huang Jun was awarded the “Top Ten Scientific Figures of the Year†by Nature in 2015; Francis of the United Kingdom in February 2016? The Crick Institute announced that the British Bureau of Artificial Insemination and Embryology has officially approved the Institute's application for editing technology for human embryonic genes; in August 2016, Lu Yu team, director of the Thoracic Oncology Unit at the West China Hospital of Sichuan University, will Launched the world's first clinical trial of CRISPR-Cas9 gene editing for lung cancer. British "Nature" magazine rated it as "leading to other ongoing genetic editing experiments in the world."
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Scientists such as Songyangzhou believe that due to the continuous advancement of gene editing technology, the world is about to enter a new era dominated by biotechnology.
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Turn the incurable disease into a "chronic disease"
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"What kind of genetic mutation causes what disease? What kind of gene can fight against what kind of virus? Gene editing technology can make humans know more about themselves and control DNA code more accurately," said Ma Wenbin.
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At present, gene editing technology has shown great prospects in the fields of disease prevention, animal and plant breeding, and animal model development.
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In the field of cancer treatment, the application prospect of gene editing is also very broad. Researchers in the United States have more research on tumors. In the treatment of leukemia, Car-T, an important technology for gene editing, has made significant progress by modifying the T cells of patients to specifically identify tumor cells for clearance. The Lu ur team, which plans to conduct gene editing for clinical trials of lung cancer, said that T lymphocytes are anti-tumor "warriors" in humans, but the T-cell encoding the PD-1 protein gene may "obstruct" its attack on tumor cells. Through the forthcoming experiment, the Lu uran team used CRISPR-Cas9 technology to genetically edit T cells, selectively knock out the genes encoding PD-1 protein in T cells, and “activate†the ability of T cells to attack tumor cells.
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These achievements and efforts have shown that genetic editing is becoming an important tool for humans to completely conquer cancer. Zhou Chenyan, a children's hematologist at Sichuan Provincial People's Hospital, said that there are three main ways to deal with cancer in clinic: surgery, radiotherapy and chemotherapy, but surgery is generally only effective for early cancer, radiotherapy and chemotherapy have large side effects, and the patient's immune system will be Caused serious damage, many patients even support the pain of radiotherapy and chemotherapy. "Genetic immunotherapy, such as genetic editing, makes up for the drawbacks of the traditional three treatments and is considered to be the biggest hope for curing cancer."
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In the field of animal and plant breeding and animal model development, many new varieties with excellent traits are emerging. Transgenic plants have become an important food source for human beings. The technology of genetically edited mice, genetically edited pigs, genetically edited dogs, and genetically edited monkeys has gradually matured, providing important support for human medical experiments and organ transplantation. Ma Wenbin said that through the "humanization" of pigs, that is, the replacement of part of the genes of pigs into human counterpart genes, humans are expected to obtain reliable sources of humanized proteins or transplant organs in the future.
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Scientists generally believe that genetic editing will bring about a "disruptive revolution" in the treatment of human diseases. In the experiment of genetic modification of human embryonic hereditary pathogenesis at Sun Yat-sen University, Huang Jun successfully repaired the genes that cause beta-type thalassemia in human embryos. Huang Jun said that many human diseases are related to genetic mutations, such as thalassemia, hereditary eye diseases, cancer, and Alzheimer's disease. Using gene editing technology, humans can "correct" the mutant genes in the future and cure these diseases.
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"Currently, human drug treatment for many deadly diseases is only to inhibit the disease and delay the deterioration. In the future, genetic editing technology will change this situation and find a new path for human beings to overcome the disease." Song Yangzhou said.
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Experts such as Han Xuan believe that in the near future, gene editing technology may make gene diagnosis and gene therapy a closed-loop service. By detecting the whole genome, physical examination, after discovering problems, integrating effective tools for gene therapy and cell therapy. It turns a disease that cannot be cured into a chronic disease that can be controlled for a long time.
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China's scientific research level has already ranked the world's "second echelon"
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At present, all countries in the world attach great importance to gene editing technology, and international competition is increasingly fierce. In this field, China's relevant scientific research level has been in the world's "second echelon" after the United States, with obvious advantages.
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The first revision of the human embryonic gene, the first genetic editing clinical trial, and the first gene knockout dog... These "world's first" fully demonstrate that the scientific research ability and scientific research level in China's genetic editing application field have reached a high level. Some Western researchers believe that in the field of CRISPR/Cas9 gene editing research, China's position is already "the world's second."
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In terms of the team reserve of scientists, China is also in the forefront. Ma Wenbin said that China's genetic editing talent reserves are among the best in the world, and there are currently more than 100 gene editing teams in China. The natural index rankings show that scientific research in China's life sciences has grown rapidly, with a 30% increase between 2012 and 2014. "This shows that a sufficient talent pool is having a profound impact on Chinese life science research, including genetic editing. The potential and explosiveness of relevant research is extremely impressive," said Ma Wenbin.
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As the world's largest genetic editing application market, China also has obvious advantages of “technology convergenceâ€. Song Yangzhou said that in recent years, some of the world's top genetic editing teams are very concerned about the Chinese market. With the evolution of applications and further market development, China is expected to become a capital and talent gathering place in the world of genetic editing.
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However, there are still many gaps in China's genetic editing field and the world's top level. Mainly in the original research is less, the core patents are less, and the future technological evolution is subject to people. The scientists interviewed said that at present, China's research on gene editing technology focuses on development or application, and its core technologies are from abroad. Foreign patent monopoly will become the bottleneck of the development of Chinese gene editing technology.
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Accelerated accumulation of knowledge will usher in a "big outbreak"
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"In the field of genetic editing research and experimentation, data accumulation is accelerating. The amount of data in a year is the sum of the past few decades. The key breakthroughs are being realized, and the technological achievements will usher in explosive growth." Ma Wenbin said.
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With the breakthrough progress of key technologies in recent years, biotechnology, especially in the field of gene editing, has become a hot spot of global academic and industrial circles. Many industry experts believe that the current development of genetic editing is in a period of great opportunities, and countries are all investing in research related to genetic editing. Knowledge accumulation has been on the eve of the outbreak. Future gene editing may make alternate breakthroughs in tool research and applied research.
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On the one hand, there is a possibility of major breakthroughs in tool research. This year, the team of Han Chunyu of Hebei University of Science and Technology announced the invention of NgAgo-gDNA technology, a new technology different from CRISPR-Cas9. Although there are still many controversies about this academic achievement, scientists generally believe that with the rapid accumulation of data and knowledge, the emergence of new tools and new technologies is “just around the cornerâ€.
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On the other hand, the application research of gene editing technology is equally broad. Wang Jian, director of the gene editing department of Huada Gene, said that gene editing technology is only partially used in genetic information, and will be extended to RNA editing and epigenetic regulation in the future. "If compared with the development of imaging, the current Gene editing is like being in the B-ultrasound and X-ray period of imaging. CT and MRI have not yet been developed, but in the future, research will be more and more in-depth." Huang Jun believes that only the current gene editing tools, There is a huge space for application development, and with the advancement of technology and tool iteration, the related application results will explode.
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Scientists suggest that in the critical period of global gene editing research, China should scientifically guide the public's objective understanding of gene editing, vigorously promote the basic research of gene editing, and further expand applied research. Of course, because genetic editing involves ethical issues, relevant research should also be strictly monitored and managed. "The journey of human beings to discover their own mysteries must be properly and strictly controlled. It is necessary to prevent scientific stagnation caused by stagnation of food, and to prevent modified genes from becoming a member of the human gene pool, and to open up Pandora, which artificially changes the course of human evolution. Magic Box '." Song Yangzhou said.