腺相关病毒载体工程研究

Recent Advances in Engineering Adeno-Associated Virus

近年来,已有3种采用重组腺相关病毒载体(Recombinant Adeno-Associated Viral Vector,rAAV)的基因疗法获批上市,且已证明这些新型疗法对遗传性罕见病有长期治疗作用。但是,rAAV作为基因疗法载体仍面临着转导效率低、载体嗜性有待提高以及会引发宿主免疫清除反应等挑战。目前,已有多项研究表明,对rAAV的载体序列进行优化,对载体衣壳进行工程改造可以改善转导效率、载体嗜性,避免宿主免疫反应并提高大规模生产的产量。本文对近年来rAAV载体工程研究方面取得的进展进行简单梳理和总结,旨在为其大规模的运用提供参考依据。

Recently,3 gene therapies based on recombinant adeno-associated viral vector(rAAV)have been approved and proved effective for treatment of genetic rare diseases.However,rAAV as a gene therapy vector still faces challenges,which include inefficient transduction,limited vector tropism and induction of immune responses against rAAV vectors and transduced cells.Currently,there have been a number of researches showing that optimization of the transgene expression cassette and capsid engineering can improve rAAV transduction efficiency,vector tropism,the ability to avoid host immune response,as well as increase large-scale productivity.This review aims to briefly summarize the progress made in rAAV vector engineering in recent years.