Division of Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill , Chapel Hill, NC , USA and.
J Drug Target. 2013 Dec;21(10):926-39. doi: 10.3109/1061186X.2013.830310. Epub 2013 Aug 22.
To improve the nuclear-targeted delivery of non-viral vectors, extensive effort has been carried out on the development of smart vectors which could overcome multiple barriers. The nuclear envelope presents a major barrier to transgene delivery. Viruses are capable of crossing the nuclear envelope to efficiently deliver their genome into the nucleus through the specialized protein components. However, non-viral vectors are preferred over viral ones because of the safety concerns associated with the latter. Non-viral delivery systems have been designed to include various types of components to enable nuclear translocation at the periphery of the nucleus. This review summarizes the progress of research regarding nuclear transport mechanisms. "Smart" non-viral vectors that have been modified by peptides and other small molecules are able to facilitate the nuclear translocation and enhance the efficacy of gene expression. The resulting technology may also enhance delivery of other macromolecules to the nucleus.
为了提高非病毒载体的核靶向递送效率，研究人员在智能载体的开发方面进行了广泛的研究，以克服多种障碍。核膜是基因传递的主要障碍。病毒能够通过特殊的蛋白成分穿过核膜，将其基因组高效地递送到细胞核内。然而，由于与后者相关的安全问题，人们更倾向于使用非病毒载体而非病毒载体。非病毒递送系统被设计为包含各种类型的成分，以在核膜的外围实现核易位。本文综述了关于核转运机制的研究进展。经过肽和其他小分子修饰的“智能”非病毒载体能够促进核易位，并增强基因表达的效果。该技术还可能增强其他大分子向细胞核的输送。
