What is gene therapy?

It is obvious that various diseases are directly or indirectly provoked by abnormalities of the gene itself or the impaired function of a gene(s). Gene therapy is a novel medical treatment in which gene(s) related to such disorders would be transferred into a patient’s body as a drug. The first gene therapy on humans was carried out in 1990 in the USA to patients suffering from adenosine deaminase (ADA) deficiency, an inheritable disease. Thereafter, it has been reported that approximately 4,000 gene therapy cases targeting inheritable disorders, cancer and AIDS etc have been carried out globally. Nowadays, the range of target disorders has been extended to such chronic disorders as rheumatism and ischemic heart diseases etc. In Japan too, gene therapy has also been carried out from 1995. In gene therapy, any therapeutic benefit can not be obtained with only the gene(s) itself alone, and a “carrier”, a vector that transports the gene into cells of the patient is absolutely necessary.