基因治疗20年

1990年9月14日美国NIH临床中心首次采用基因治疗成功治愈腺苷脱氨酶（ADA）基因缺陷而患重度联合免疫缺损和免疫系统功能低下疾患，至今已整整20年，其发展迅速，从单纯的重组技术导入基因DNA发展到了涵盖DNA和RNA两个干预水平、和基因上调（如基因增补、矫正、置换等）及下调（基因失活）的两大策略，近年来的进展使得基因治疗登入《Science》杂志2009年度十大科学进展，我国在基因治疗领域诞生了第一个上市药物，有10多个制剂临床前和多个在临床研究。基因治疗在遗传病治疗中具备巨大潜力，已经成为当代生命科学中最有前景的研究方向之一。

The 20 Years of Gene Therapy

It was totally twenty years now from Sep. 14, 1990 when the first successful case of gene therapy. The treatment was for adenosine deaminase (ADA) deficiency patient which born with severe combined immunodeficiency (SCID) and without functioning immune systems in NIH Clinical Center of USA. So far a lot of progress had been made in gene therapy. It was developed from pure gene recombinant technology for DNA transfering to both DNA and RNA molecular levels, and from deficient gene upregulating such as augmentation, correction, replacement, etc. to both gene expression up-regulating and down-regulating (gene inactivation) strategies. Gene therapy was the year news of Science in 2009. In China, the first gene therapy medicine was approved for the therapy, and a dozen reagents were on the study, as well several ones on clinical trails. Gene therapy has a potential for trestment the hereditary disease, becomes the promising biotechnolgy in life science.