视网膜色素变性患者复明的希望  

Hope for the patients with retinitis pigmentosa

作　　者：尹卫靖[1] 王丽娅[1] 王薇[2] 潘峰[3] 

机构地区：[1]河南省眼科研究所,郑州450003 [2]北京大学第三医院眼科北京大学眼科中心,100083 [3]美国纽约大学眼科、神经科学和生理系,10016

出　　处：《中华实验眼科杂志》2011年第2期97-100,共4页Chinese Journal Of Experimental Ophthalmology

摘　　要：视网膜色素变性（RP）是一种遗传性视网膜疾病，其典型的临床表现为早期视杆细胞的退行性病变以及视锥细胞胞体相对长期的存活。目前实验研究证实，通过腺相关病毒载体和慢病毒载体将微生物型的光敏感通道蛋白channelrhodopsin-2或halorhodopsins导入RP模型鼠的视锥细胞胞体或其他细胞，可以使这些细胞获得光反应并激活视网膜传导通路，向视觉中枢传递视觉信息。因此，这为RP患者的复明带来了希望。Retinitis pigmentosa （RP） is a hereditary disease characterized by the progressive loss of photoreceptor ceils. Disease pathology primarily affects rod photoreceptor cells first but light-insensitive cone photoreceptor bodies can survivor longer. Delivered by adeno-associated viral vector or lentiviral vector, expression of microbial-type rhodopsin, channelrhodopsin-2 or archaebacterial halorhodopsin in the survived retinal ceils can substitute for the native phototransduction cascade in mouse models of retinitis pigmentosa. It can also restore light sensitivity and activate all retinal cone pathways and drive sophisticated retinal circuit functions. Thus, expression of channelrhodopsins or halorhodopsin might be a potential strategy for the restoration vision of patients with RP.

关 键 词：视网膜色素变性 基因疗法 视杆细胞 视锥细胞 腺病毒载体 慢病毒载体 光敏感通道蛋白 

分 类 号：R774.1[医药卫生—眼科]