非病毒载体在基因治疗中的发展与应用

Advances of Non-Viral Delivery Systems Applied in Gene Therapy

构建基因载体并建立安全高效的基因载体体内投递系统是基因治疗的关键。基因载体分病毒与非病毒载体。病毒载体转染高效,已应用于临床,但仍面临安全性低的问题。非病毒载体制备简单、安全性高、潜力大;然而,转染效率低的问题限制了其临床应用。基因治疗领域的研究者一直致力于优化非病毒载体投递系统,提高转染效率,已经出现可应用于临床的基因投递产品。文章旨在回顾非病毒基因载体的研究进展及其临床应用前景。

Establishment of safe and effective gene vectors and in vivo delivery systems are crucial for gene therapy. The vectors for gene therapy comprise viral and non-viral vectors. Of which the viral vectors have been widely applied in clinical trials, due to their highly transduction efficiency; while they still encounter many potential safety concerns. In contrast, the non-viral vectors are safe and easy to prepare gene vehicles. However, the insufficient transfection efficiency limits their clinical application. Many efforts have been devoted into optimizing the delivery systems to enhance the gene transfer efficiency of non-viral vectors, a few of which are currently undergoing clinical trials. In this review, the recent advances of non-viral gene delivery systems and their clinical prospects were summarized.