改造HBV作为基因治疗载体

HBV具备改造作为肝靶向性基因治疗载体的基本条件---天然嗜肝特异性,能够在肝细胞内持续复制、反复感染,病毒本身对细胞没有明显的细胞毒性;能够携带外源基因并被包装成病毒颗粒;能够介导外源基因的转移和表达。但由于基因结构复杂,目前改造的HBV载体均存在载容量小、复制包装效率低及安全性差等缺点。就近年相关研究进展进行综述。

The Progress in Reconstructing Hepatitis B Virus as Gene Therapeutic Vector

Hepatitis B virus (HBV) is naturally provided with the prerequisite for being transformed as the liver-targeting gene therapeutic vector because of its hepatotropism specificity. It specifically infects the liver and continuously replicates in the hepatocytes without obvious cytotoxicity by itself. Moreover, it has also the capability of carrying, transferring and expressing the gene of interest, which is ultimately encapsidated into the viral particles. However, it is a pity that there are many shortcomings in the present developed HBV-based vectors such as the poor capability of carrying long foreign gene, the low efficiency of replication and encapsidation, the potential risk of immunogenicity etc, which all result from the complex genomic structure of HBV. The major progress in studying HBV vector for the liver-targeting gene therapy is outlined.