腺相关病毒载体介导视网膜疾病的基因治疗研究进展  

The development in the studies of AAV-mediated gene therapy in retina

作　　者：邹绚[1] 睢瑞芳[1] 董方田[1] 

机构地区：[1]中国医学科学院北京协和医学院北京协和医院眼科,100730

出　　处：《中华眼科杂志》2011年第11期1049-1052,共4页Chinese Journal of Ophthalmology

摘　　要：视网膜疾病分子机制研究的不断深入，促进了视网膜疾病基因治疗的进展。腺相关病毒（AAV）的毒性和免疫原性均较低，外源基因表达稳定，可以转染多种分裂期和静止期细胞，因此成为治疗视网膜疾病的有效载体。在不同的动物模型和临床试验中，已有多项研究结果证实AAV作为载体治疗视网膜疾病的安全性和有效性。目前AAV在动物疾病模型中已经进行了介导抗血管生成蛋白、神经营养因子、抗凋亡因子的表达和基因替代治疗的研究，取得了令人满意的效果。但是，AAV也存在携带外源基因能力偏小的问题，需要进一步研究并解决之。Significant progress in understanding the molecular basis of retinal disorders has led to the development of gene therapies for treatment of these diseases. Adeno-assoeiated virus （AAV） is a useful vector for the treatment of retinal diseases clue to its low toxicity and dimmunogenicity, ability to transducer both dividing and non-dividing cells, and stable transgene expression. A variety of animal studies and clinical trials have proved the safety and effectivity of retinal AAV-mediated gene therapy. AAV-mediated gene therapy, such as anti-angiogenic proteins, neurotrophie factors, anti-apoptosis factors were studied in animal disease models, and the results were satisfactory. However, the main drawback of AAV vectors is its relatively small packaging capacity, which needs further improvement.

关 键 词：依赖病毒 转染 基因疗法 视网膜疾病 

分 类 号：R77[医药卫生—眼科]