In pursuit of new developments for gene therapy of human diseases

Gene therapy aims at transferring a therapeutic gene into human somatic cel ls in order to treat a disease. Originally addressed to hereditary genetic disorders, gene therapy has found therapeutic applications in cancer, infec tious diseases and degenerative disorders, particularly those of the nervou s system. Although gene transfer into humans has been demonstrated in sever al clinical trials, with more than 300 currently underway worldwide, there is still no single outcome that undoubtedly showed a consistent benefit for the patient. Nevertheless, the expectations for gene therapy are still hig h, and the prospects of future clinical success are increasing together wit h the growing of the field. The development of better delivery systems spec ifically tailored to individual diseases, with sustained expression of the therapeutic gene in the appropriate cells, will in the end make possible tr ue therapeutic applications of human gene transfer. (C) 1949 Elsevier Scien ce B.V. All rights reserved.