[Present status and future prospects of human gene therapy].
Gene therapy is defined as the treatment of diseases by the transfer of genes into cells. This concept was originally emerged as the definitive therapy for genetic diseases. However, acquired diseases such as cancer and AIDS are now being considered for its potential targets. The first clinical trial of human gene therapy began in 1990 at the NIH for treating a patient with fatal severe combined immunodeficiency caused by adenosine deaminase deficiency. Gene therapy of cancer has also initiated at many research hospitals. Currently, more than 100 patients have already been treated by this innovative therapeutic strategy in the US. In Japan, the Expert Committee on Gene therapy was set up in the council on Science and the Public Health and Welfare in 1991. Recently, the committee released the guidelines of gene therapy. It is thought that the first gene therapy in Japan is not far off.
Department of Biochemistry and Molecular Biology, Nippon Medical School, Tokyo, Japan.
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D005544:Forecasting; D015316:Genetic Therapy / Q000639:trends*; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000628:therapy; D009369:Neoplasms / Q000628:therapy; D016511:Severe Combined Immunodeficiency / Q000628:therapy