Gene transfer technology in therapy: Current applications and future goals

Gene therapy has attracted much interest since the first submissions of pha se I clinical trials in the early 1990s, for the treatment of inherited gen etic diseases. Preliminary results were very encouraging and prompted many investigators to submit protocols for phase I and phase II clinical trials for the treatment of inherited genetic diseases and cancer. The possible ap plication of gene transfer technology to treat AIDS, cardiopathies, and neu rologic diseases is under evaluation. Some viral vectors have already been used to deliver HIV-1 subunits to immunize volunteers who are participating in the AIDS vaccine programs in the USA. However, gene delivery systems st ill need to be optimized in order to achieve effective therapeutic interven tions. The purpose of this review is to summarize the latest achievements i n improving gene delivery systems, their current application in preclinical studies and in therapy, and the most pressing issues that must be addresse d in the area of vector design.