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基因治疗肝脏疾病的新策略已引起高度关注,在肝病的基因治疗中,最关键的是如何将治疗基因特异性地导入肝细胞中并适当表达。在过去的二十多年里,受体介导的基因给药系统广泛用于肝靶向基因递送,但一些非病毒载体的基因传递效率不高。本文综述了目前最常用的非病毒载体,包括其理化性质、优点和局限性,基因递送作用机理以及修饰后在肝靶向基因治疗中的应用,并综述了在肝细胞基因传递中常用的电穿孔技术和流体力学注射法等物理方法以及如何实现其最优化的转染率。
The new strategy of gene therapy for liver diseases has drawn much attention. In the gene therapy of liver diseases, the key is how to specifically transduce the therapeutic gene into hepatocytes and appropriately express it. For more than two decades, receptor-mediated gene delivery systems have been widely used for liver-targeted gene delivery, but gene delivery by some non-viral vectors is not efficient. This review summarizes the most commonly used non-viral vectors, including its physical and chemical properties, advantages and limitations, gene delivery mechanism and modification of liver-targeted gene therapy, and reviewed the commonly used in liver cell gene delivery of electricity Physical methods such as perforation techniques and hydrodynamic injections, and how to optimize their transfection rates.