Zou Xuan, Sui Rui-fang, Dong Fang-tian
Department of Ophthalmology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing 100730, China.
Zhonghua Yan Ke Za Zhi. 2011 Nov;47(11):1049-52.
Significant 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 immunogenicity, 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.
在理解视网膜疾病分子基础方面取得的重大进展，已推动了用于治疗这些疾病的基因疗法的发展。腺相关病毒（AAV）因其低毒性和免疫原性、能够转导分裂和非分裂细胞以及稳定的转基因表达，成为治疗视网膜疾病的有用载体。各种动物研究和临床试验已证明视网膜AAV介导的基因疗法的安全性和有效性。在动物疾病模型中研究了AAV介导的基因疗法，如抗血管生成蛋白、神经营养因子、抗凋亡因子，结果令人满意。然而，AAV载体的主要缺点是其相对较小的包装容量，这需要进一步改进。
