Research Center for Stem Cell Engineering, National Institute of Advanced Industrial Science and Technology (AIST), 1-1-1 Higashi, Central 4, Tsukuba, Ibaraki, 305-8562, Japan.
Curr Gene Ther. 2012 Oct;12(5):410-6. doi: 10.2174/156652312802762518.
Gene delivery/expression vectors have been used as fundamental technologies in gene therapy since the 1980s. These technologies are also being applied in regenerative medicine as tools to reprogram cell genomes to a pluripotent state and to other cell lineages. Rapid progress in these new research areas and expectations for their translation into clinical applications have facilitated the development of more sophisticated gene delivery/expression technologies. Since its isolation in 1953 in Japan, Sendai virus (SeV) has been widely used as a research tool in cell biology and in industry, but the application of SeV as a recombinant viral vector has been investigated only recently. Recombinant SeV vectors have various unique characteristics, such as low pathogenicity, powerful capacity for gene expression and a wide host range. In addition, the cytoplasmic gene expression mediated by this vector is advantageous for applications, in that chromosomal integration of exogenous genes can be undesirable. In this review, we introduce a brief historical background on the development of recombinant SeV vectors and describe their current applications in gene therapy. We also describe the application of SeV vectors in advanced nuclear reprogramming and introduce a defective and persistent SeV vector (SeVdp) optimized for such reprogramming.
自 20 世纪 80 年代以来,基因传递/表达载体一直被用作基因治疗的基本技术。这些技术也被应用于再生医学中,作为将细胞基因组重编程为多能状态和其他细胞谱系的工具。这些新研究领域的快速进展以及将其转化为临床应用的期望,促进了更复杂的基因传递/表达技术的发展。自 1953 年在日本分离以来,仙台病毒(SeV)已被广泛用作细胞生物学和工业中的研究工具,但最近才开始研究将 SeV 作为重组病毒载体的应用。重组 SeV 载体具有各种独特的特性,例如低致病性、强大的基因表达能力和广泛的宿主范围。此外,该载体介导的细胞质基因表达在应用中具有优势,因为外源基因的染色体整合可能不理想。在这篇综述中,我们介绍了重组 SeV 载体发展的简要历史背景,并描述了它们在基因治疗中的当前应用。我们还描述了 SeV 载体在高级核重编程中的应用,并介绍了一种优化用于这种重编程的缺陷和持续 SeV 载体(SeVdp)。