近日，国际药物控制释放领域权威杂志Journal of Controlled Release刊登了中科院上海生命科学研究院健康科学研究所张晓玲研究员的最新综述文章“Uptake mechanisms of non-viral gene delivery”，文章中，研究者阐述了在非病毒基因输送体系的研究进展。
基因（分子）治疗是人类重大疾病的治疗可能取得突破的重要领域。然而，传统上用于基因输送的病毒载体在作为药物使用时，其安全性问题一直以来备受质疑，使得其难以在临床上广泛应用。一些化学合成的非病毒基因载体尽管在安全性上优于病毒载体，但由于其作为基因药物难以在体内有效的将基因输送到靶细胞，并且难以被细胞所高效吸收，因而基因输送效率一直是其走上临床的瓶颈问题。
近年来，健康所骨科细胞与分子生物学组硕士研究生向晟楠等在张晓玲研究员的指导下，在非病毒基因输送体系的研发及骨相关疾病的治疗领域进行了一系列的研究工作。此篇综述结合研究成果，集中介绍了非病毒基因输送系统被细胞内吞并吸收的生物学机理以及相关的研究进展，为生物学家、药物学家未来合作设计出更加高效的非病毒基因输送系统提供了重要的理论参考。（生物谷Bioon.com）
doi:10.1016/j.jconrel.2011.09.093
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Uptake mechanisms of non-viral gene delivery
Shengnan Xianga, Haijun Tonga, c, Qin Shic, Julio C. Fernandesc, Tuo Jind, Kerong Daia, b, Xiaoling Zhanga,
Non-viral gene delivery is currently a hot subject for its relative safety and simplicity of use; however, it is still far from being ideal enough to be clinically used for its comparatively lower efficiency than viral gene delivery. To improve the efficiency of non-viral gene delivery needs a comprehensive understanding of the uptake mechanisms. Macromolecules are internalized into cells by a variety of mechanisms, and their intracellular fates are usually relevant with the uptake pathways. The uptake pathways of non-viral gene complexes are usually determined by not only the gene/carrier interaction but also by the interaction between complexes and target cells. The best-characterized uptake pathway is the so-called clathrin-mediated endocytic pathway. However, there are numerous updates of knowledge about endocytic pathways and even non-endocytic pathways in recent years with the development of novel technologies for tracking and inhibiting. In this review, we will try to sort out our current understanding of the uptake mechanisms of non-viral gene delivery. In addition, factors for pathway selection are summarized in the third section. Finally, the useful inhibitors or tools for the study of these pathways will also be concluded in the last section.
