慢病毒载体及其介导的RNA干扰在基因治疗中的应用研究

Study of the application of lentiviral vector mediated RNA interference on gene therapy

主要就慢病毒载体及其介导的RNA干扰技术在基因治疗中的应用研究进行了综述,并对其在该领域具有的广阔前景进行了展望.慢病毒载体作为一种新型的载体,可有效地将携带的目的基因导入宿主细胞,并将其整合到宿主细胞基因组中,从而使目的基因得以持久稳定地表达.该载体因具有高感染性、高表达效率及不易诱发宿主免疫反应等优点,已成为基因治疗研究中的一个重要工具.RNA干扰技术可以特异性抑制或关闭特定基因的表达,因此该技术可广泛应用在基因功能探究和恶性肿瘤治疗等领域.由慢病毒载体介导的RNA干扰技术能持久、稳定、特异性地抑制各类细胞中特定基因的表达,在病毒感染、肿瘤等疾病的基因治疗中被广泛应用,成为生物医学领域的新热点.

It was summarized the application of lentiviral vector and its related RNA interference on gene therapy,an outlook to its broad prospects for application was given. Lentiviral vector,a kind of new retroviral vector,could deliver exogenous genes into host cells and integrate them into the host genomes,leading to the persistent and stable expression of exogenous genes. Because of its high efficiency of infection and expression as well as low host immune reaction,lentiviral vector had been an important tool in gene therapy research. RNA interference（RNAi）technology could specifically knock down and turn off the expression of target genes, therefore,it had been widely used in studying gene function and cancer therapy. Moreover,lentiviral vector mediated RNAi could stably inhibit the expression of specific genes. Thus,it was expected to provide new methods for gene therapy.