Howe Steven J, Chandrashekran Anil
Molecular Immunology Unit, Wolfson Centre for Gene Therapy, UCL Institute of Child Health, London, UK.
Methods Mol Biol. 2012;891:85-107. doi: 10.1007/978-1-61779-873-3_5.
Vectors derived from the Retroviridae family have several attributes required for successful gene delivery. Retroviral vectors have an adequate payload size for the coding regions of most genes; they are safe to handle and simple to produce. These vectors can be manipulated to target different cell types with low immunogenicity and can permanently insert genetic information into the host cells' genome. Retroviral vectors have been used in gene therapy clinical trials and successfully applied experimentally in vitro, in vivo, and in utero.
源自逆转录病毒科的载体具备成功进行基因传递所需的多个特性。逆转录病毒载体对于大多数基因的编码区域而言，具有足够的有效载荷大小；它们易于操作且生产简单。这些载体可经操控以靶向免疫原性低的不同细胞类型，并且能够将遗传信息永久插入宿主细胞的基因组。逆转录病毒载体已用于基因治疗临床试验，并在体外、体内和子宫内实验中成功应用。
