中文摘要：

如何降低免疫反应是重组腺相关病毒（rAAV）基因药物面临的重大挑战。研究表明减少载体使用剂量能够降低rAAV基因药物的免疫反应，但是减少载体使用剂量同时也会降低靶基因的表达量，甚至可能达不到治疗效果。本文重点阐述如何在减少载体使用剂量的同时，保持基因表达强度和药效。尤其侧重讨论采取给药途径优化、病毒载体改造、启动子选取在增强基因治疗效果中的应用。

英文摘要：

How to reduce immune response is an unprecedented challenge for rAAV gene medicine. Recent studies suggested that lowering dosage of the vector used could reduce immune response caused by rAAV gene medicine. Nevertheless, it would also decrease the transgene expression, leading to failure of gene treatment. It is therefore important to take appropriate steps to maintain high gene expression level and pharmacodynamic, while the dosage of rAAV used is reduced. Here, steps to enhancing gene therapy, such as optimization of the administration, reconstruction of the viral vector and selection of the promoter, are discussed in order to achieve maximum outcome.