近日，國際藥物控制釋放領(lǐng)域權(quán)威雜志Journal of Controlled Release刊登了中科院上海生命科學(xué)研究院健康科學(xué)研究所張曉玲研究員的最新綜述文章“Uptake mechanisms of non-viral gene delivery”,，文章中,，研究者闡述了在非病毒基因輸送體系的研究進(jìn)展。
基因（分子）治療是人類重大疾病的治療可能取得突破的重要領(lǐng)域,。然而,，傳統(tǒng)上用于基因輸送的病毒載體在作為藥物使用時，其安全性問題一直以來備受質(zhì)疑,，使得其難以在臨床上廣泛應(yīng)用,。一些化學(xué)合成的非病毒基因載體盡管在安全性上優(yōu)于病毒載體，但由于其作為基因藥物難以在體內(nèi)有效的將基因輸送到靶細(xì)胞,，并且難以被細(xì)胞所高效吸收,，因而基因輸送效率一直是其走上臨床的瓶頸問題。
近年來,，健康所骨科細(xì)胞與分子生物學(xué)組碩士研究生向晟楠等在張曉玲研究員的指導(dǎo)下,，在非病毒基因輸送體系的研發(fā)及骨相關(guān)疾病的治療領(lǐng)域進(jìn)行了一系列的研究工作。此篇綜述結(jié)合研究成果,，集中介紹了非病毒基因輸送系統(tǒng)被細(xì)胞內(nèi)吞并吸收的生物學(xué)機(jī)理以及相關(guān)的研究進(jìn)展,，為生物學(xué)家,、藥物學(xué)家未來合作設(shè)計出更加高效的非病毒基因輸送系統(tǒng)提供了重要的理論參考。（生物谷Bioon.com）
doi:10.1016/j.jconrel.2011.09.093
PMC:
PMID:
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.
