中国生物工程杂志

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.