反转录病毒基因治疗载体及其安全性评价

Retroviral Vectors in Gene Therapy and its Safety Assessment

反转录病毒载体是目前基因治疗中应用最广泛的基因运载工具之一,具有能稳定整合入宿主细胞、持久表达目的基因、感染细胞范围广、基因容量较大等优点,但其生物安全性还存在争议。在简要介绍反转录基因治疗病毒载体的基础上,对反转录病毒载体中外源目的基因的表达调控及反转录病毒载体的安全性评价进行了综述。

Retroviral vectors have become one of the most extensively used vehicles for gene therapy. The advantages that retroviral vectors offer include their abilities to stably integrate into the genomic DNA of the recipient cells, to permanently express the transduced genes, to transduce a variety of cell types, to accommodate large genes of interest. However, the biosafety of retroviral vectors is concerned and requires improvement. In this review, the progress of the study on retroviral vectors, regulation system for improving gene expression and safety assessment were discussed.