摘要/Abstract

摘要： 基因药物的细胞内高效输送是基因治疗走向临床转化的关键环节。基因等生物大分子药物往往借助载体以内吞的方式进入细胞，入胞后若不能及时从内涵体 / 溶酶体中逃逸则极易被降解失活。光化学内化作用（photochemical internalization，PCI）作为一种新兴的光促药物输送技术，利用光照下光敏剂分子产生活性氧破坏生物膜，促进内涵体 / 溶酶体膜逃逸，从而避免溶酶体中酶对药物的破坏，将生物大分子等药物有效递送到细胞质中。近年来，该技术在基因等大分子药物的细胞内有效可控输送方面取得了一定成效，为基因治疗从实验室向临床应用的转化提供了可能。该文综述了 PCI 的作用机制及其在基因输送中的应用概况和研究进展。
关键词: 基因输送, 光化学内化作用, 光敏剂, 活性氧, 内涵体 / 溶酶体逃逸
Abstract:
The translation of gene therapy from bench to bedside depends on efficient intracellular gene delivery. The macromolecular biologics such as gene combined with vectors tend to enter into the cells by means of endocytosis, where the biologics may encounter the risk of degradation in endolysosome. Recently, photochemical internalization (PCI) has emerged as a promising technique to overcome endo-lysosomal sequestration, which utilizes photosensitizer and light resulting in reactive oxygen species at sub-lethal level to destruct biofilm and facilitate intracellular drug delivery. In this article, the mechanism of PCI technology and its development for gene delivery were reviewed, which can provide the scientific basis for the possible utilization of PCI to solve the problem of endo-lysosomal escape in gene delivery.
Key words: gene delivery, photochemical internalization (PCI), photosensitizer, reactive oxygen species, endo-lysosomal escape



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