Department of Bioorganic Chemistry, Wrocław University of Technology, Wybrzeze Wyspiańskiego 27, PL 50-370 Wrocław, Poland.
Pharmacol Rep. 2009 Nov-Dec;61(6):993-9. doi: 10.1016/s1734-1140(09)70160-4.
Gene therapy has become a promising technique for the treatment of cancer. Nevertheless, the success of gene therapy depends on the effectiveness of the vector. The challenge of a gene carrier is to deliver exogenous DNA from the site of administration into the nucleus of the appropriate target cell. Polymer-based vectors are biologically safe, have low production costs and are efficient tools for gene therapy. Although non-degradable polyplexes exhibit high gene expression levels, their application potential is limited due to their inability to be effectively eliminated, which results in cytotoxicity. The development of biodegradable polymers has allowed for high levels of transfection without cytotoxicity. For site-specific targeting of polyplexes, further modifications, such as incorporation of ligands, can be performed. Most expectations have been addressed to polyplexes architecture according it dynamic response with the microenvironment.
基因治疗已成为治疗癌症的一种有前途的技术。然而，基因治疗的成功取决于载体的有效性。基因载体的挑战在于将外源 DNA 从给药部位递送到适当靶细胞的细胞核中。基于聚合物的载体具有生物安全性、生产成本低，是基因治疗的有效工具。尽管不可降解的聚电解质复合物表现出高的基因表达水平，但由于其不能有效消除，导致细胞毒性，其应用潜力有限。可生物降解聚合物的发展允许在没有细胞毒性的情况下进行高水平的转染。为了对聚电解质复合物进行特定部位的靶向，还可以进行进一步的修饰，如加入配体。大多数期望都集中在聚电解质复合物的结构上，根据其与微环境的动态响应来进行。
