Liu D, Knapp J E
Department of Pharmaceutical Sciences, University of Pittsburgh School of Pharmacy, Pittsburgh, PA 15261, USA.
Curr Opin Mol Ther. 2001 Apr;3(2):192-7.
The need for a safe and efficient method for gene delivery has stimulated the recent emergence of vectorless methods (e.g., naked DNA) as promising alternatives to the available viral- and non-viral-based systems. Among these methods, hydrodynamics-based gene delivery through systemic injection of DNA offers a convenient, efficient and powerful means for high-level gene expression in mice. This review deals with the background and progress made so far with this new gene delivery method, the potential mechanism underlying the hydrodynamics-based DNA transfer into cells and its potential applications towards gene function analysis, protein expression and gene therapy in whole animals.
对安全有效的基因递送方法的需求,促使了无载体方法(如裸DNA)的出现,作为现有基于病毒和非病毒系统的有前景的替代方法。在这些方法中,通过全身注射DNA进行基于流体动力学的基因递送,为在小鼠中实现高水平基因表达提供了一种方便、高效且强大的手段。本文综述了这种新的基因递送方法的背景和迄今取得的进展、基于流体动力学的DNA转入细胞的潜在机制,以及其在全动物基因功能分析、蛋白质表达和基因治疗方面的潜在应用。