Jakobsson Johan, Lundberg Cecilia
Department of Experimental Medical Research, CNS Gene Therapy Unit, Section for Neuroscience, Wallenberg Neuroscience Center, Lund University, 221 84 Lund, Sweden.
Mol Ther. 2006 Mar;13(3):484-93. doi: 10.1016/j.ymthe.2005.11.012. Epub 2006 Jan 3.
Lentiviral vectors have been used extensively as gene transfer tools for the central nervous system throughout the past decade since they transduce most cell types in the brain, resulting in high-level and long-term transgene expression. This review discusses some of the recent progress in this field, including preclinical gene therapy experiments in disease models, development of regulated vectors, and the application of siRNA's using lentiviral vectors. We also describe some of the features that make lentiviral vectors a likely candidate for human gene therapy in the brain.
在过去十年中,慢病毒载体已被广泛用作中枢神经系统的基因转移工具,因为它们能转导大脑中的大多数细胞类型,从而实现高水平和长期的转基因表达。本文综述了该领域的一些最新进展,包括在疾病模型中的临床前基因治疗实验、调控载体的开发以及使用慢病毒载体的小干扰RNA(siRNA)的应用。我们还描述了一些使慢病毒载体成为大脑人类基因治疗可能候选者的特征。