Palù G, Bonaguro R, Marcello A
Institute of Microbiology, University of Padova, Italy.
J Biotechnol. 1999 Feb 5;68(1):1-13. doi: 10.1016/s0168-1656(98)00134-5.
Gene therapy aims at transferring a therapeutic gene into human somatic cells in order to treat a disease. Originally addressed to hereditary genetic disorders, gene therapy has found therapeutic applications in cancer, infectious diseases and degenerative disorders, particularly those of the nervous system. Although gene transfer into humans has been demonstrated in several 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 high, and the prospects of future clinical success are increasing together with the growing of the field. The development of better delivery systems specifically tailored to individual diseases, with sustained expression of the therapeutic gene in the appropriate cells, will in the end make possible true therapeutic applications of human gene transfer.
基因治疗旨在将治疗性基因导入人体体细胞以治疗疾病。基因治疗最初针对遗传性基因疾病，现已在癌症、传染病和退行性疾病，尤其是神经系统疾病中发现了治疗应用。尽管在多项临床试验中已证明基因可导入人体，目前全球有300多项临床试验正在进行，但仍没有一个结果能毫无疑问地显示对患者有持续一致的益处。然而，对基因治疗的期望仍然很高，随着该领域的发展，未来临床成功的前景也在增加。开发专门针对个别疾病量身定制的更好的递送系统，使治疗性基因在适当的细胞中持续表达，最终将使人类基因转移真正的治疗应用成为可能。
