腺相关病毒载体的研究进展

Progress in research on adeno-associated virus vectors

腺相关病毒(adeno-associated virus,AAV)是目前应用较为广泛的病毒载体之一。AAV载体作为基因治疗的工具,具有较高的生物安全性和稳定性、较低的免疫原性、较广的宿主范围、较强的特异性、可长时间表达外源基因等特点,因此,在基因治疗的研究过程中备受关注。本文就AAV的发现与生物学特性、重组AAV(r AAV)载体的设计、AAV载体在临床基因治疗中的应用及其可能引发的不良反应作一综述,以期为AAV载体今后的研究方向提供参考。

Adeno-associated virus(AVV)is one of the widely used viral vectors.As a tool for gene therapy,AAV vector has high biological safety,high stability,low immunogenicity,wide host range and high specificity,which may be used for long-term expression of foreign genes.Therefore,it is favored by researchers in gene therapy.This article reviews the discovery,biological characteristics,design of recombinant AAV(r AAV)vector,application of AAV vector in clinical gene therapy and possible adverse reactions so as to provide a reference for further research.