高效基因靶向递送系统研究进展

Efficient and targeted delivery systems for gene therapy:research advances

基因药物是一类重要的生物大分子药物,靶点明确,专属性强,在重大疾病的治疗中显现出十分巨大的潜力。然而,尽管有多项基因药物进入临床研究,目前全球范围内批准上市的基因药物仍屈指可数。其主要原因在于基因药物的体内递送面临易降解、靶向性差、入胞难等一系列问题。已有多种精巧的药物递送系统被成功用于基因药物的体内递送,提高了基因药物的靶向性和治疗效果。本文对基因药物体内靶向递送研究面临的问题,以及高效递送系统在克服体内外递送屏障方面的最新进展进行综述。

Therapeutic genes are a class of important biomacromolecules,which have great potential in treating severe diseases. Despite of the fact that several nucleic acid-based drugs have entered clinical trails,the number of approved therapeutic genes is quite limited. The main reason is that the in vivo delivery of therapeutic gene is faced with a series of problems such as easy degradation,poor targeting,difficulty in cellular internalization. To address these issues,a variety of sophisticated drug delivery systems have been successfully designed and applied in the delivery of therapeutic genes,which have improved the targeting and therapeutic effects.In this paper,we review the difficulties in the in vivo delivery of therapeutic genes and the latest progresses in overcoming the delivery barriers.