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.

abstractsSignificant progress in understanding the molecular basis of retinal disorders has led to the development of gene therapies for treatment of these diseases.Adeno-associated virus (AAV) is a useful vector for the treatment of retinal diseases due 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,neurotrophic 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.