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Research Progresses of Gene Therapy


ZHANG Yansong, CHEN Lijiao, ZHANG Ting, MA Yue, LI Shangang*

(Institute of Primate Translational Medicine, Kunming University of Science and Technology, Yunnan Key Laboratory of Primate Biomedical Research, Kunming 650000, China)
Abstract:

After 30 years of unremitting efforts by scientists, the concept of gene therapy has finally been applied to the clinical medicine, and has gradually become an important means for treating genetic diseases, infectious diseases and tumors, etc. Gene therapy offers more options for the diseases that cannot be treated with traditional therapies. Meanwhile, the rapid development in the gene editing field and the accumulated experience in the use of delivery vectors have also greatly promoted the progress of gene therapy. To date, the EMA (European Medicines Agency) and the U.S. FDA (Food and Drug Administration) have approved the marketing of six gene therapy products. Gene therapy has good prospects as a new treatment method, but it still faces many challenges to continue to develop into mainstream therapies. This paper reviews the development of gene therapy, focuses on the current state of technology development and clinical application in the field, and summarizes and prospects the safety and ethics of gene therapy.