Vector systems for prenatal gene therapy: principles of retrovirus vector design and production.
Vector systems for prenatal gene therapy: principles of retrovirus vector design and production.
复制标题
DOI：
10.1007/978-1-61779-873-3_5
复制
发表时间：
2012-01-01
期刊：
影响因子：
--
通讯作者：
Chandrashekran, Anil
中科院分区：
文献类型：
--
作者：
Howe, Steven J;Chandrashekran, Anil
来源于逆转录病毒科的载体具有成功基因递送所需的几个属性。逆转录病毒载体对于大多数基因的编码区具有足够的有效载荷大小;它们操作安全且生产简单。这些载体可以被操纵以靶向具有低免疫原性的不同细胞类型，并且可以将遗传信息永久地插入宿主细胞的基因组中。逆转录病毒载体已被用于基因治疗的临床试验，并成功地应用于体外，体内和子宫内的实验。
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.