《生命科学》 2013, 25(6): 595-600

2型腺相关病毒作为基因治疗载体的研究进展

陈如意，陈素峰，周　丹，许　健^*

(浙江中医药大学生命科学学院，杭州 310053)

摘　要：摘　要：2 型腺相关病毒(adeno-associated virus， AAV-2) 属于细小病毒科依赖病毒属，因具有无致病性、宿主范围广、目的基因持久表达等优点而成为当前基因治疗中最有潜力的病毒载体之一。然而，AAV-2 载体也存在着免疫原性弱、靶向性不强及转导效率不高等问题，但是随着AAV 在分子病毒学领域的不断研究，其不足也找到了可能的解决思路和方案。综述了AAV-2 的生物学特点及作为基因治疗载体的优势，并着重介绍了其在基因治疗应用中存在的不足及应对策略。
关键词：2 型腺相关病毒；载体；基因治疗

Advances on research of AAV-2 vector in gene therapy

CHEN Ru-Yi， CHEN Su-Feng， ZHOU Dan， XU Jian^*

(College of Life Science， Zhejiang Chinese Medical University， Hangzhou 310053， China)

Abstract: Abstract: Adeno-associated virus type 2 (AAV-2) belongs to the family Parvoviridae of the genus Dependovirus. AAV-2 is a potential delivery vehicle for gene therapy application， which possesses some features including the lack of pathogenicity， wide range of host， and persistent target gene expression. However， its host immune response， weak targeting property and low transduction efficiency also limit its application in gene therapy. Meanwhile， novel approaches have been developed to mitigate the drawbacks with the further research progress on the AAV-2 in the virology field. This article describes the biological characteristics and superiority of AAV-2， and highlights its limitations and coping strategies as a carrier in gene therapy application.
Key words: AAV-2; vector; gene therapy