ФC31整合酶系统在基因治疗中的应用  

Application of ФC31 Integrase System in Gene Therapy

作　　者：李振海[1] 马晴雯[1] 

机构地区：[1]上海市儿童医院、上海交通大学附属儿童医院、上海交通大学医学遗传研究所,上海市200040

出　　处：《医学分子生物学杂志》2012年第4期281-285,共5页Journal of Medical Molecular Biology

基　　金：国家重点基础研究发展计划（973计划）（No.2010CB529902）

摘　　要：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。Gene therapy offers promising prospects for the treatment of diseases. As a non-viral vector system, ФC31 integrase system has many advantages, such as high efficiency, site-specific- ity and low toxicity. ФC31 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, advan- tages and disadvantages of ФC31 integrase system, as well as its biological safety.

关 键 词：基因治疗 ФC31整合酶 体内应用 离体应用 安全性 

分 类 号：R394.3[医药卫生—医学遗传学] R34[医药卫生—基础医学]