Takatsuki K, Obaru K, Yoshimura K, Matsushita S
Second Department of Internal Medicine, Kumamoto University School of Medicine.
Nihon Rinsho. 1996 Jan;54(1):233-41.
Genetic manipulation of somatic cells may be of therapeutic value in a variety of infectious diseases, particularly in human immunodeficiency virus (HIV) infection. Stable insertion of "resistance genes" into cells, susceptible to HIV, could reduce the viral burden in infected individuals and potentially retard the characteristic progressive immune dysfunction. Alternatively, ectopic expression of genes that encode viral antigens, might induce potent antiviral immune responses and form the basis for novel prophylactic and therapeutic vaccines. While laboratory studies have proved that the approach works in principle, preclinical and clinical studies will be necessary to evaluate the therapeutic benefits of such gene-based therapies. Currently, more than 400 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, gene therapy for ADA has been approved. It is thought that the first gene therapy against HIV infection in Japan is not far away.
对体细胞进行基因操作在多种传染病治疗中可能具有重要价值,尤其是在人类免疫缺陷病毒(HIV)感染方面。将“抗性基因”稳定插入对HIV敏感的细胞中,可降低感染者体内的病毒载量,并有可能延缓典型的进行性免疫功能障碍。另外,编码病毒抗原的基因的异位表达可能会诱导强大的抗病毒免疫反应,并构成新型预防性和治疗性疫苗的基础。虽然实验室研究已证明该方法原则上可行,但仍需要进行临床前和临床研究,以评估此类基因疗法的治疗效果。目前,美国已有400多名患者接受了这种创新治疗策略的治疗。在日本,1991年在科学与公共卫生和福利委员会中设立了基因治疗专家委员会。最近,针对腺苷脱氨酶(ADA)的基因治疗已获批准。人们认为,日本首例针对HIV感染的基因治疗已为期不远。