腺相关病毒(AAV)在治疗B型血友病的应用研究进展

Research Progress of Adeno-Associated Virus(AAV)in the Treatment of Hemophilia B

腺相关病毒(AAV)是一种常用于基因传递、表达的载体,具有良好的安全性,较低的免疫源性与可以稳定表达等特点.B型血友病(Hemophilia B),又叫做乙型血友病,是一种由凝血因子Ⅸ基因缺陷而引起的X染色体隐性遗传疾病.近年来,国内外研究者在利用AAV进行基因治疗临床相关疾病的研究中取得了新的进展,给B型血友病患者带来了治愈的希望.本文就腺相关病毒的生物学特性,目前用于基因治疗的腺相关病毒载体类型及其在治疗B型血友病中的研究进展做一综述.

Adeno-associated viruses(AAV)are vectors commonly used for gene delivery and expression,which has the advantages of good safety,low immunogenicity,stable expression and so on.Hemophilia B is a bleeding disorder inherited from X-linked recessive inheritance caused by defects in the factor IX(FIX)gene.Recent clinical trials of gene therapy using AAV vectors demonstrated encouraging effects and relative safety.In this article,the biological characteristic of AAV,the types of vectors used for gene therapy,and the application in the treatment of hemophilia B will be summarized.