纳米粒子介导的脉络膜新生血管基因治疗

Nanoparticle-mediated gene therapy for choroidal neovascularization

脉络膜新生血管（CNV）是多种致盲眼病包括年龄相关性黄斑变性等的主要致盲原因。由于疾病的自身特性以及眼屏障的存在使得CNV的治疗成为一个棘手的问题。目前基因治疗是一种新的治疗方向，随着分子材料学的进展，纳米粒子成为一种极具前景的基因治疗载体。纳米粒子，特别是聚乳酸（PLA）及与聚乙醇酸（PGA）的共聚物（PLGA）纳米粒子具有良好的组织相容性及生物可降解性，可安全长效地介导体内、外视网膜色素上皮细胞的特异性转染。目前采用纳米粒子携带治疗基因通过偶联特异性配体等方式，主要通过玻璃体腔或全身静脉注射两种给药途径，可安全有效地抑制实验性CNV。（国际眼科纵览，2012，36：346—352）

Choroidal neovascularization （CNV） is the main cause of severe visual impairment in many blindness-leading diseases, including age-related macular degeneration （AMD）. At present, treatment for CNV is still a challenging work since drug delivery may be limited to the retinal pigment epithelium （RPE） and choroid because of several formidable barriers. In this article, we reviewed the properties of nanoparticles, and recent substantial progress in the development of nanoparticles as a targeted vector of gene therapy for CNV, which is a promising approach to provide safe and long-term gene expression to inhibit ex- perimental CNV. （ Int Rev Ophthalmol, 2012, 36 ： 346-352 ）