Engineering technology

Gene Accurate Regulation and Medical Technology Based on Nucleic Acid Substances

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Improve the efficiency of CRISPR/Cas9 gene editing technology, reduce miss efficiency, and realize safe and efficient in vivo application; How to use mRNA to realize the development of nucleic acid vaccines and drugs and solve the key scientific problems in mRNA nucleic acid technology (stability, large-scale preparation, high-efficiency expression, safe transportation, etc.) are the key scientific problems in this field.

Question Background:

Looking back on the pharmaceutical process of human beings, from the most classic small molecule drugs to antibody drugs, China lags behind the West for decades or hundreds of years from basic research to clinical drug development. Nucleic acid technology, including RNA interference technology, CRISPR/Cas9 gene editing technology and mRNA-based nucleic acid technology, which has been studied and arisen in the past 20 years, represents the development trend and direction of biomedical technology in the future. Among them, the discovery of RNA interference won the Nobel Prize in Physiology or Medicine in 2006. It took only 8 years from its discovery in 1998 to the award. CRISPR/Cas9 gene editing technology is also considered to be an important discovery that will win the Nobel Prize. In these emerging technological fields, China must and fully have the ability and potential to keep pace with western developed countries in this new generation of biomedical technology following small molecules and antibodies. In the next 30 years, we must surpass and lead in some fields. However, the premise to achieve the previous goal is to carry out top-level design and planning from a strategic perspective in this field, and to implement and implement it strongly.


Latest developments:

The development technology of small interfering RNA drugs based on RNA interference has matured, and the first small nucleic acid drug will be marketed in the first quarter of 2018. The key technical problems that limit its deeper and wider drug development are how to solve its safe and efficient in vivo transportation and how to achieve long-term stability. At present, the delivery tissue that has been basically solved is liver. In other tissues and organs, there is no delivery technology that can really support drug development.

At present, new progress is being made in gene editing technology based on CRISPR, how to improve editing efficiency, reduce or avoid miss, and how to develop gene editing or silence technology derived from CRISPR. Scientists around the world are investing great energy and resources in this field to research and develop mature technologies that can eventually move towards industry.

In mRNA-based nucleic acid technology, Because it can realize the expression of any gene of interest and avoid the potential gene integration risks brought about by the use of viral vectors in traditional gene therapy, foreign countries are currently carrying out relatively early technological research and development in full swing. Innovative vaccine preparation based on mRNA or expression of new drug proteins can realize effective prevention and control of diseases.

On the whole, the above technologies can realize precise regulation of gene expression at the nucleic acid level, representing the most advanced innovation technology in the field of biomedical technology and the future development direction. At present, the world is continuously making new progress. China needs to catch up, surpass and lead in this field, and needs deeper and higher-level research urgently.

Significance:

Through in-depth and forward-looking research on the above nucleic acid technologies, China can be helped to realize and lead the way in the next 30 years in the next generation of biomedical technologies that have not been realized in the fields of small molecular compounds and monoclonal antibodies. Accurate prevention and control of major diseases affecting human health can be realized from the gene level.

In this field, the corresponding planning, design and research on national strategies such as "Healthy China 2030" and "Integration of Defense and Civilian Technologies" are of great significance to China's social development and national rejuvenation.


Referral Information

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Chinese Society of Biophysics
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Author's Introduction

Huang Yuanyu
Unit:Associate Researcher, Beijing University of Technology Professional Title: