AAV在基因治疗中的靶向修饰研究进展

Progress in Targeting Modification of Viral Vector AAV in Gene Therapy

安全、有效、具有靶向性的病毒载体是基因治疗药物在临床上得以应用的关键。AAV是微小病毒科的一种,它能以其低的免疫原性及广泛的宿主性对人及灵长类进行感染,并且经过改造后的AAV病毒能更有效的靶向性特定组织及肿瘤细胞。重点对AAV病毒载体的衣壳蛋白基因工程修饰、转录调控修饰和转录后microRNA干扰表达修饰及衣壳蛋白化学修饰靶向机理,以及改造方法进行介绍。修饰后的AAV能改善其感染引起的性免疫反应、转染效率和肿瘤靶向性。

Safe,effective and targeting viral vector is the key of gene therapy for clinical application.Adeno-associated virus（AAV） belong to the family of Parvoviridae.It is a small virus which can infects humans and some other primate species with low-immunogenicity and extensive host cell.Beside,modificated AAV can target the specifical tissus and tumor cells effectively.In this article,introduce the principles and methods of the genetic engineering modification of AAV capsid proteins,the transcription regulation modification of target gene expression,the post-transcription regulation modification of microRNA interference in target genes translation,and the chemic modification of AAV capsid proteins were introduced.The modificated AAV can improve the immune response from self-assemble of virus,its transfection ratio,its tumor target.