腺相关病毒衣壳蛋白修饰的研究进展

Research advances in capsid protein modification of adeno-associated virus

腺相关病毒(adeno-associated virus,AAV)介导的基因治疗在单基因遗传性疾病的临床治疗中取得了突破性进展。通过衣壳蛋白修饰来优化AAV载体,获得具有细胞靶向性、高转导能力和低或无免疫原性的AAV载体成为当前基因治疗研究的重点之一。目前,研究者主要通过合理设计、定向进化和化学修饰方法对AAV衣壳进行修饰、改造。本文综述了AAV的生物学特性和有关AAV衣壳修饰的新近研究进展,并讨论了该领域所面临的挑战。

Adeno-associated virus(AAV)-mediated gene therapy has led to a breakthrough in the clinical treatment of single-gene disorder.Optimizing AAV vector through capsid modification to obtain AAV vector with cell targeting,high transduction efficiency and low or no immunogenicity has become one of the focuses of gene therapy research.At present,AAV capsid is mainly modified and reconstructed by rational design,directed evolution and chemical modification.Here,we review the biological characteristics of AAV and the recent research advances in the modification of AAV capsid,and discuss the challenges in this field.