Cai Yujia from the system Biomedical Research Institute of Shanghai Jiaotong University believes that China has taken the lead in the research and development of gene therapy drugs. As early as 2003, China has approved the world's first gene therapy drug "Jin re Sheng" for cancer treatment. After that, it entered a period of relative silence. He concluded that at present, the development of gene therapy in China focuses too much on car-t cell therapy, while little attention is paid to the drug research and development of classic gene therapy. According to incomplete statistics, there are more than 100 car-t companies, but less than five classic gene therapy companies.

 

 

 

The opposite is true abroad. In 2012, glybera, the world's first gene therapy drug for genetic diseases, was born in the European Union. In 2016 and 2017, the global pharmaceutical giant GlaxoSmithKline's gene therapy strimvelis realized a gene therapy for severe immunodeficiency for the first time. Novartis car-t alleviated relapsed and refractory acute lymphoblastic leukemia (all) in children and adolescents more than 80% of the unprecedented cases.

 

 

 

China's R & D in this field is advancing. Professor Yan Guangmei of pharmacology of Sun Yat sen University and his team's latest achievements on anti-cancer of natural a virus M1 oncolytic virus have increased the anti-tumor activity of M1 virus by 3600 times. They found that the new oncolytic virus M1 has the characteristics of selectively killing a variety of tumor cells. This is the first oncolytic virus to achieve precise treatment, which will greatly improve the therapeutic effectiveness and efficiency of oncolytic virus. Oncolytic virus therapy is an innovative strategy for tumor targeted therapy. It selectively infects tumor cells with natural or genetically modified viruses, and then achieves the purpose of killing tumors through various mechanisms, but it is harmless to normal cells. The prepared lyophilized powder injection of oncolytic virus M1 can be stored at 37 ℃ for three weeks and at 2 ℃ ~ 8 ℃ for one or two years.

 

 

 

"Only one course of treatment, 35% of the mice liver cancer disappeared, completely cured, no recurrence." Yan Guangmei introduced his experimental results in genetic modification at this forum. "In another 10 years, at most 20 years, cancer will no longer be a frightening disease, and we can control it well.".

 

 

 

At present, there are more than 7000 rare diseases in the world. Although the patient population is relatively small, 95% of the diseases have no effective treatment. 50% of the patients are children, and more than 80% of the rare diseases are related to gene mutation. It is reported that researcher Li Dali and his team from the school of life sciences of East China Normal University have recently launched the development and Application Research of monobasic gene technology. Using the Abe system formed by adenosine deaminase and cas9 protein, the model of multiple genetic diseases has been constructed efficiently and accurately, which is expected to achieve the curative effect of once treatment and lifelong cure.

 

 

 

Children are the biggest victims of genetic diseases. On average, there is one case of congenital deafness in every 1000 newborns, and more than 70% of them are caused by genetic factors. However, there is no progress in the treatment of genetic deafness. Tao Yong, an otolaryngologist in the Ninth People's Hospital of Shanghai Jiaotong University Medical College, has been engaged in the treatment of deafness. The discovery of CRISPR / cas9 and the improvement of editing efficiency in vivo provide a new tool for gene therapy of inner ear. Tao Yong's research confirmed that CRISPR / cas9 can edit gene in the small and complex inner ear of mice, and restore the hearing of mice to normal. In the future, the cure of congenital deafness is no longer a dream.

 

 

 

Experts say that the recovery of gene therapy technology will change the direction of medical treatment in the future, and the era of gene and cell therapy has come.