中枢神经系统组织特异性启动子在基因治疗中的研究及其进展

将外源基因有效的导入神经细胞并使其靶向、高效和长期稳定的表达是目前基因治疗神经系统疾病的主要靶点。随着研究的深入,越来越多的组织特异性启动子已被用于调控目的基因在靶细胞或靶组织中的表达,成为基因治疗研究领域的热点之一。现综述目前常用的几种中枢神经系统组织特异性启动子在基因治疗中的研究及其进展。

Progress in the use of cell-specific promoters for gene therapy in the CNS

Restricting transgene expression to specific cell types and maintaining effective and long-term expression are major goals for gene therapy in the central nervous system. The use of cell-specific promoters has been applied for these purposes, They may provide a method to target the specific expression of transgene in different tissues and cell types. In this article, progress in the use of several cell-specific promoters for gene therapy in the CNS has been viewed.