关键词: font-size:medium, ">基因治疗；基因补充疗法；基因抑制疗法；基因调控疗法

Abstract: The original concept of gene therapy was to introduce DNA sequences containing normal gene through vectors into cells to correct diseases caused by defects in that gene. Because this concept was very intuitive and easy to be accepted， and the genetic diseases caused by numerous gene mutations were largely untreatable， gene therapy had received great attention as soon as it was proposed. With the rapid development of genetic engineering since the 1970s， gene therapy had entered clinical exploration from laboratory research. The first gene therapy clinical trial was completed in 1990， entering a period of rapid development. A decade later， global gene therapy research slowed down markedly as a patient died unexpectedly in a gene therapy trial in the United States. The resurgence of gene therapy US FDA-approved for the Luxturna (2017) treatment of Leber sysdroms and Zolgensma (2019) for treatment of spinal muscular atrophy (SMA) is marked by the which was a sign that effective and expensive genetic drugs have finally become a powerful weapon in the hands of clinicians to the pain of previously helpless patients. In addition to gene replacement therapy， gene editing therapy for different clinical needs， gene inhibition therapy based on antisense oligonucleotide and small interfering RNA， gene regulatable therapy based on gene switch， and other gene therapy methods are in comprehensive research and development. This paper summarizes the development process of gene therapy， its development inrecent years， and its further advancement and application in the future.

Key words: font-size:medium, ">Gene therapy；Gene editing therapy； Gene inhibition therapy；Gene regulatable therapy