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脑源性神经营养因子(BDNF)是一种由脑组织合成并广泛分布于中枢神经系统的小分子碱性蛋白.研究发现,BDNF对视网膜神经节细胞(RGCs)、视网膜感光细胞(RPCs)和视网膜色素上皮(RPE)细胞均有保护、营养及抗凋亡作用.视网膜色素变性(RP)是由于RPCs及RPE细胞凋亡引起的视网膜退行性疾病.RP动物模型证实了BDNF的长期给药对于RP的治疗价值.然而BDNF在体内的半衰期较短,且无法跨越血-视网膜屏障由循环系统输送到视网膜,这给BDNF用于RP的治疗带来了挑战.为了使BDNF在眼内可以稳定持续地释放,多种新型给药方式已被尝试,包括基因工程技术、细胞移植技术、高分子材料缓释系统及滴眼液等.本文就BDNF对RP治疗的研究现状及BDNF的新型给药方式做一综述.“,”Brain-derived neurotrophic factor (BDNF) is a kind of small molecule basic protein mainly synthesized in cortical areas and widely expressed in central nervous system.According to recent studies,BDNF plays a role in protection,nutrition and anti-apoptosis to retinal cells,including retinal ganglion cells (RGCs),retinal photoreceptor cells (RPCs) and retinal pigment epithelium (RPE) cells.Retinitis pigmentosa (RP) is a degenerative fundus disease due to the progressive degeneration of the RPCs in the retina.Mammalian modules verified the value of long-term BDNF in RP therapy.However,short half-life in vivo and failing to pass the blood-retina barrier become the obstacles to deliver BDNF intraocular,which bring challenges to BDNF applying in clinic.To make BDNF release intraocular stable and sustained,novel delivery system for BDNF have been tasted,such as gene engineering techniques,cell plantation techniques,sustained-released polymer delivery system and eye-drops.This article focuses on current research status of BDNF to RP treatment and its novel drug delivery system.