Gene editing technology has continuously brought surprises to mankind in recent years, and it has become a key technology supporting many advanced therapies such as organ transplantation and cell therapy.

As the source of the next generation of biotechnology revolution, researchers believe that gene editing technology will enable amazing innovations in medicine, agriculture and many other fields, but related ethical challenges and technological innovation still need to be overcome.

Underlying technological innovation to be broken through

The world's first heart xenograft patient survived for two months after receiving a heart from a genetically modified pig, a miracle in medical history. Using the gene-editing technology CRISPR-Cas9, US researchers have transplanted ten genetically modified pig organs into humans for the first time, and the results show that the approach is working, at least for a while.

Last month, a US research team claimed to have cured a female AIDS patient for the first time using a cutting-edge stem cell transplant. Along this line, researchers hope to use genetic modification to find a cure for AIDS and eradicate the virus once and for all.

Crispr-Cas9, the earliest gene editing technology based on bacterial immune mechanisms, has shown great potential in human drug therapy. It has now become the basis for the treatment of genetic diseases such as sickle cell anemia, and will provide precise treatment for more diseases in the future. plan.

my country attaches great importance to the research and development of gene editing, and has listed it as a key research goal during the "14th Five-Year Plan" period. In January of this year, the state issued a safety management specification for gene editing products, paving the way for the promotion of gene editing applications.

At the two sessions this year, how to realize the underlying innovation breakthrough of gene editing technology has attracted the attention of members of the Chinese Academy of Sciences.

Cao Xiaofeng, member of the National Committee of the Chinese People's Political Consultative Conference, academician of the Chinese Academy of Sciences, and researcher of the Institute of Genetics and Developmental Biology of the Chinese Academy of Sciences, said: "Although my country is at the forefront of the world in the research and development and application of gene editing technology for animals and plants, the original patents are seriously absent, and most of the core patents and underlying technologies are Monopolized by Western countries led by the United States, industrial security is facing serious challenges, and accelerating the promotion of basic research on genome editing is an urgent need for national development and strategic layout."

Cao Xiaofeng said that gene editing is developing rapidly in two aspects: in basic research, including mechanism research of gene editing tools, model establishment, original innovation research and development, and optimization of existing editing tools; in terms of technical application, accelerating research results The transformation and application of gene editing technology will achieve a deeper technological leap, promote the implementation of the industry, and build a bio-economy with gene editing technology as the pillar.

At present, many units in my country are accelerating the establishment of gene editing technology centers and laboratories to promote the industrialization of gene editing technology in my country into the fast lane. However, Cao Xiaofeng believes that the layout of the domestic gene editing field mostly focuses on product-driven technology research and development and transformation applications, lack of basic research on gene editing tools and original innovation of underlying technologies, and it is difficult to completely reverse the passive situation that core technologies and industrial development are controlled by others.

In this regard, she suggested strengthening the strategic scientific and technological strength in the field of gene editing, creating a "Gene Editing Basic Discipline Research Center", and increasing capital investment in basic research; focusing on multiple research and application dimensions such as animals and plants, microorganisms, major diseases and modern agriculture , carry out in-depth research on the molecular mechanism of gene editing, structural analysis of programmable nucleases, new tool mining, delivery and editing new technology research and development; promote the output of original and subversive basic research results, and accelerate the transformation of research results and the industrial application of technology.

Ethical regulations need to be improved

At the same time, the scientific community and global governments are still exploring and seeking solutions on how to use innovative biotechnology to benefit mankind within an ethical framework. In the process, some radical attempts have also raised doubts.

For example, He Jiankui, a former professor at the Southern University of Science and Technology in China, used gene editing technology to edit the fetuses of AIDS patients. However, this rash behavior has caused huge controversy in the world scientific community due to ethical disputes and falsification of experimental ethics approval procedures.

But this still cannot stop the wheel of scientific progress. In April last year, Chinese researchers published a blockbuster paper in the journal Cell, creatively injecting human stem cells into the embryos of non-human primate cynomolgus monkeys for the first time. - Monkey chimeric embryos survived for nearly 20 days. This achievement announced a major breakthrough in the research of human-monkey chimeric embryos, and the in vitro organ cultivation of stem cells has taken a step forward.

In the long term, researchers also hope to use these chimeras to study early human development, develop disease models, screen for potential new drugs, and create cells, tissues or organs that can be transplanted and, one day, new hearts and kidneys. Rescue patients in need of transplants.

Professor Ji Weizhi, an academician of the Chinese Academy of Sciences and the Institute of Primate Translational Medicine of Kunming University of Science and Technology, who is in charge of the above research, believes that biotechnology, including gene editing and gene fusion, still needs to make breakthroughs in ethics and innovation.

Taking xenotransplantation as an example, Ji Weizhi told the First Financial Reporter: "Ethically, there should be regulations on related xenotransplantation, and technically, it is necessary to improve the survival rate of xenogeneic organs and reduce immune rejection."

He believes that xenotransplantation may also be realized in China in the future, and some restrictions on research ethics will be revised to adapt to the development of science.

Artificial Organs University of Maryland