Gene editors create miracles and hope to cure hemophilia

Release date: 2017-06-30

On June 27th, the media reported that Japanese researchers have successfully treated mouse hemophilia using gene editing technology, and it is expected to cure human hereditary hemophilia in the future.

In recent years, similar research results have emerged in an endless stream, which has encouraged scientists to see the infinite possibilities of curing diseases. The gene editing with great potential has also triggered the infinite imagination of the masses. Can we finally break through the shackles of the flesh and transform into the omnipotent heroes to save the evil world? However, returning to the reality of bones, gene editing technology has been controversial since its birth. It involves many ethical issues such as human ethics, law, social influence and regulatory rules. It has already surpassed the simple technical boundary and became the moral bottom line for all human beings. The ultimate test.

In 2015, Professor Huang Jun of Sun Yat-Sen University successfully used the CRISPR technology to successfully modify the human embryo gene for the first time. This result immediately caused great controversy in the scientific and ethical circles at home and abroad. Can he edit the human germline genes and break through the ethical restrictions? The focus of the debate. After two years of exploration and thinking, in February of this year, the Human Gene Editing Research Committee finally released a heavyweight report, which made detailed and rigorous scientific norms for the application of human gene editing technology.

At the 8th International Forum on Clinical and Translational Hospitals in China, Prof. Zeng Fanyi, Director of the Shanghai Institute of Genetic Diseases, gave a speech on "Genetic Editing: Science, Ethics and Management" of the American Academy of Sciences, and made a professional interpretation of the main purpose of the report. . The forum was hosted by the Sino-US Scientific Committee on Clinical and Translational Medicine and the Global Doctors Organization. The Shell Society participated in the conference as a support media. The contents of Professor Zeng Fanyi's speech are now organized as follows:

1, gene editing application human experiment

The occurrence of many human diseases is related to gene sequences, and the discovery of DNA double helices has opened up biological progress. Human beings have always had the dream of genetic modification. They have made countless attempts over the years to achieve precise manipulation of DNA. However, reality faces many difficulties and obstacles.

The traditional genetic modification process is complex, based on the orientation modification of embryonic stem cells and genetic recombination, which is usually laborious and time-consuming and costly. There is data that a genetic modification of the traditional method costs an average of 5,000 US dollars. Recently, the hot genetic editing has made significant progress in technology, enabling genetic modification in a short period of time, saving time and effort, and being more precise, efficient and flexible than previous strategies. To this end, the 2011 “Genome Editing Nuclease” was named an annual research method by Nature, and the new CRISPR/CAS9 system was named one of the top ten scientific and technological advances by UNESCO in 2015.

Gene editing technology finely modifies the DNA sequence of the gene to understand the nature of life, growth and development mechanisms, understand the mechanism of disease occurrence and the purpose of treating diseases. The novel gene editing technology is mediated by artificial nuclease, specifically recognizes and cleaves the target DNA double-strand, and stimulates the repair mechanism to achieve gene-oriented transformation. Compared with the past, the target has high target efficiency, low construction cost and wide application range.

The three representative technologies, represented by ZFN, TALEN, and CRISPR/CAS, each have their own characteristics:

At present, the use of CRISPR technology has been successful in different species, and different editing effects such as site-breaking and gene knockout have been achieved in different cell line materials, and CRISPR-mediated gene regulation is not only genomic sequence cutting, but also There are functions such as gene expression inhibition and gene expression activation. For this reason, CRISPR is called a weapon to manipulate molecular genetics and epigenetics, and its many benefits make it a new favorite of the scientific community.

In fact, these three gene editing technologies have been applied in many aspects of human gene therapy since 2005.

More importantly, gene editing technology will be applied to human trials. In July 2016, Nature published heavy news. Chinese scientists from West China Hospital led the world and launched the world's first CRISPR human trial. In December, Nature reported that gene editing technology has been successfully used in China for immunotherapy. The National Institutes of Health also launched the first CRISPR human trial in the United States at the end of 2016 to explore the potential of technology and save more lives.

2, in addition to prevention, treatment of diseases, genetic editing is not allowed

Along with the advancement of technology, there are many other problems that come with it. We should set up a sound system early, including ethics and control systems, to ensure good development along the expected path from the source.

From the three major goals of genetic editing research, including basic research, somatic cell intervention, and intervention in the reproductive system. Basic research can focus on cellular, molecular, biochemical, genetic, or immune mechanisms, including effects on reproduction and development, and disease development, and response to treatment; clinical applications for somatic genome editing only affect the patient itself, similar to existing ones. Using gene therapy to treat and prevent disease does not inherit or affect the next generation; and interventions in the reproductive system that aim to change the genome will not only affect the next generation, but also some of them, and may change species. Etc., so the ethical problems caused by stem cell or germ cell intervention are greater for the treatment of human diseases.

This has caused a series of problems, such as the existing technology of somatic gene editing is still not perfect, there is a need to do risk assessment for off-target effects. While germline gene editing affects the next generation, although it can provide some families with the best choice to block the spread of disease, it is also highly controversial and goes beyond many ethical boundaries.

There is also a very important ethical issue, if genetic editing is beneficial as a means of good health or disease prevention, such as using genetic editing to improve muscle tissue in patients with muscular dystrophy. But if you use this technology to enhance muscle function and increase the limits of human muscle strength to meet or exceed the abilities of mortal people, this will go beyond the public health category, will increase social injustice, trigger social contradictions and pressures of comparison, the public is edited by genes. The “enhancement” response will become more and more uneasy, and it will inevitably have a series of negative effects. Therefore, public discussion reveals that regulatory risk and benefit analysis is necessary, and no genome editing other than treatment, disease prevention, and disability should be performed at this stage.

Public participation is also very important, and widespread public participation and input, as well as a reassessment of health social benefits risks, is one of the key conditions for the approval of clinical trials to date. Public communication and consultation mechanisms have been established in foreign countries, and public consultation is required extensively. The US Recombinant DNA Advisory Committee must provide a public discussion of gene therapy sites, hosted by the Federal Social Council, and open to the public. Other countries, such as Britain and France, may also have formal voting or hearing mechanisms to ensure acceptance of different views.

Human genome editing is a double-edged sword. There are scientific and ethical issues at different levels. It should be controlled to promote its proper use and avoid misuse. In the context of the cultural, political and legal context of each country, it is the significance of regulation to clarify the restrictions and safeguards used by the technology.

3. Strict supervision and revision of genetic diseases

There are US and international policy discussions abroad, as well as for emerging genetic editing. In 2015, a group of researchers and ethics workers met with CRISPR/Cas9 developers in California to advocate for the society to explore the nature of human genome editing and provide guidelines for its acceptable use; at the end of 2015, in Washington, USA The National Academy of Sciences and Academy of Medical Sciences, the Chinese Academy of Sciences, and the Royal Society of the United Kingdom jointly held the "Gene Editing Summit" on the development, current status, and potential applications and risks of genetic editing techniques, as well as ethical, legal, social, and regulatory rules. Conducted a multi-party discussion. A consensus was reached at the meeting that, given the many benefits that genetic technology would bring to humans, research on gene editing should be encouraged and supported under strict preconditions. In addition, a series of influential international conventions, review bodies or organizations have also expressed their views on further regulating the use of genetic editing.

On February 14, 2017, the Human Gene Editing Research Committee, affiliated with the American Academy of Sciences and the American Medical School, released Human Genome Editing: Science, Ethics, and Goverance after more than a year of research. In this report, the United States The 22 well-known scientists, doctors, ethicists, and policy scholars at the Academy of Sciences and the American College of Medicine believe that we should allow scientists to edit human sperm, eggs, or early embryos using sophisticated genetic editing techniques under strict supervision. Major genetic diseases such as sickle cell anemia and cystic fibrosis.

One of the most important things to talk about is "genetic diseases." Globally, there are thousands of genetic diseases caused by single-gene mutations, which together affect 5~7% of the world's population. It can not ignore the emotional and economic burden of patients' families. For patients with lysosomal storage disease, Duchenne muscular dystrophy, sickle cell anemia, cystic fibrosis, etc., if you want to have healthy children, editing the germ cells may be the only way right now. . The Reporting Committee believes that if it is proven to be safe and reliable, genetic editing should be allowed to correct mutations that will be passed on to the next generation.

Another important keyword is "strict supervision." In order to avoid the emergence of gray areas, the Commission has put forward a series of requirements: only when preventing major diseases, and there is no other reasonable means to try, can we try genetic editing of germ cells; scientists must strictly confirm that these edited genes lead to Major diseases, or the incidence of major diseases is greatly increased; credible preclinical and clinical trials are needed to assess the risks and benefits of this approach, ensuring the health and safety of participants; researchers must Several generations of follow-up analysis to understand long-term risks; genetic editing of germ cells can only be used to prevent disease, and can not be used to increase non-health factors such as population intelligence, height, strength.

The management principles of the human genome editor quote the words of the famous French microbiologist and chemist Louis Pasteur: "Science has no national boundaries, because knowledge is the heritage of mankind." In this principle, although science is global, But it is carried out within various political systems and cultural norms. It is very important, but also difficult, to formulate a principle that can transcend differences and differences and adapt to cultural diversity.

The Human Genome Management Principles have seven important aspects: promoting well-being, transparency, proper care, being accountable to science, respecting individuals, fairness and cross-border cooperation.

First, the principle of promoting well-being is only to promote human health and well-being, to treat or prevent diseases, to minimize the risk of early high uncertainty, and to reasonably balance risks and benefits.

The second principle is transparency, which requires open and shared information to stakeholders in an accessible and understandable way, and promises to disclose information in the most timely and timely manner;

The third is proper care, which refers to careful and dedicated care of patients enrolled in the study or receiving clinical care;

The fourth is responsible for science, requiring basic standards and clinical research in accordance with international and professional standards, maintaining the highest standards, conducting high-quality experimental design and analysis, and properly reviewing and evaluating data;

Fifth, we have always respected individuals, acknowledged the importance of personal choice, and respected and advocated individual decisions;

Sixth, the principle of fairness, including the rights and obligations of equitable distribution research, benefits broadly and equitably from the clinical application of human genome editing;

Finally, it is transnational cooperation, respecting the different cultural backgrounds, political backgrounds and historical backgrounds of countries, coordinating countries to regulate, setting standards and procedures, and implementing cross-border cooperation and data sharing.

4. Domestic must make up for regulatory and ethical gaps

Finally, the status quo of Chinese gene editing is introduced. The research and development of Chinese gene editing is very fast. So far, the number of papers and patents are among the highest in the world. Scientists in China have taken the lead in using gene scissors CRISPR technology to establish important models of rats and pigs and genetic modification models of economic animals. In April 2015, a team led by Huang Jun, an associate professor at Sun Yat-sen University, published a paper in the journal Protein and Cell, using CRISPR/Cas9 technology to edit non-viable human embryos, attempting to modify genes in human embryos that may induce beta thalassemia. . According to Nature, Huaxi Hospital launched the world's first human application of CRISPR technology. The first patient received these T-cells modified by CRISPR technology, and the patient's living condition is very good.

According to New Scientist, May 30, 2017, with the first genetic modification experiment using CRISPR technology in the human body to eliminate the oncogenic HPV virus, more than 20 genetically edited human trials will be carried out, among which Part of it is carried out in China, and the future use of CRISPR technology for genetic editing will continue to heat up.

Therefore, China's technological development is at the forefront. In our research progress, there is a need for consensus between the world and the bio-industry community, because genetic editing will bring revolutionary changes to basic research and translational medicine, and is the core of the next generation of biotechnology. Although China's genetic editing research is developing very fast, it also faces certain challenges. We need to make up for the regulatory and ethical gaps, and we need to refer to a series of foreign regulations. China's genetic editing technology and research applications, the current relatively disorderly layout, and the relatively weak current ethics and regulatory legal aspects must be changed.

To this end, we encourage the creation of genetic editing technologies with self-intellectual property rights, as well as the development of relevant policies on the translation of genetic editing results as soon as possible, and the promotion of genetic editing techniques for the study of major diseases and treatments. It is hoped that while genome editors bring spring to human health and disease treatment, China can play a leading role in this field, both in terms of scientific ethics and management.

Source: Shell Society

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