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用病毒作为载体携带功能基因感染视网膜以达治疗目的,这种基因治疗方法为某些遗传性视网膜病如视网膜色素变性的治疗开辟了新途径。应用腺病毒或腺相关病毒载体,经视网膜下间隙或玻璃体注射,可使报告基因在视网膜细胞中有效表达。腺病毒介导的基因表达起效早,持续时间短;腺相关病毒介导的基因表达起效晚,持续时间长。病毒载体中所含启动子的不同,其基因导入的靶组织也不同。含巨细胞病毒启动子的病毒载体可将基因转移至视网膜色素上皮细胞;应用视紫红质启动子则转移至视细胞。
The use of the virus as a vector to carry a functional gene infection of the retina for therapeutic purposes, this gene therapy for some hereditary retinal diseases such as retinal pigmentosis opened up a new way. Application of adenovirus or adeno-associated virus vector, the subretinal space or vitreous injection, the reporter gene can be effectively expressed in retinal cells. Adenovirus-mediated gene expression early onset, duration is short; adeno-associated virus-mediated gene expression onset of effect for a long time. The viral vectors contain different promoters, and the target tissues into which the genes are introduced are also different. The viral vector containing the cytomegalovirus promoter can transfer the gene to the retinal pigment epithelium; the rhodopsin promoter is transferred to the visual cell.