ФC31整合酶系统在基因治疗中的应用

摘要

Gene therapy offers promising prospects for the treatment of diseases. As a non-viral vector system, OC31 integrase system has many advantages, such as high efficiency, site-specificity and low toxicity. OC31 integrase can mediate the integration of functional genes into the genome of target cells, leading to a stable and long-term expression of the therapeutic genes in varieties of tissues and organs, both in vivo and ex vivo. This review focused on the mode, application, advantages and disadvantages of OC31 integrase system, as well as its biological safety.%基因治疗是一种极具应用前景的疾病治疗方法.作为一种非病毒载体系统,ФC31整合酶系统凭借其高效性、位点特异性与低毒性的特点,在基因治疗的应用中具有无可比拟的优势.ФC31整合酶可以介导治疗基因整合到靶细胞基因组并稳定、长期表达,目前已经在多种组织和器官的体内、离体研究中取得成功.文章着重阐述ФC31整合酶的作用机制、应用实例及优缺点,并评估了其安全性.