中文摘要：

重组腺相关病毒（rAAV）具有免疫原性低、在各种细胞中介导的转基因表达时间长等优点,将成为最广泛应用的基因治疗载体之一。rAAV基因药物临床研究历经16年,其有效性和安全性已得到充分证实,但其准确、可靠的定量测定标准方法仍未建立。目前采用的rAAV滴度测定方法按照原理可分为生物测定和物理测定两大类。生物测定主要评估载体传递转基因的能力,包括转导和感染滴度测定。物理测定则基于其物理性质,可细分为病毒颗粒和基因组滴度测定。本文对现有rAAV滴度测定方法的基本概念、原理、优点、缺点和适用范围等作一详细介绍。

英文摘要：

Recombinant adeno-associated virus（rAAV） is recognized as one of the most widely used gene therapy vector in the future because it elicits minimal immune responses and mediates long-term transgene expression in a variety of cell types.Sixteen years have passed since the first clinical trial of rAAV,which have demonstrated the efficacy and safety of rAAV.However,there is still no standard method for measuring rAAV titer accurately and reliably.Presently available methods for rAAV titer assay can be divided into biological and physical assays.The biological assays seek to estimate the ability of the vector to deliver the transgene of interest and including transduction assays and infection assays.The physical assays are based on the physical properties of rAAV,such as virus particle number or packaged genomes quantity.In this review,the basic concept,principle,advantage,disadvantage and their potential applications of each type of assays for rAAV titer are discussed in detail.