Liu Shuai, Gao Yongsheng, A Sigen, Zhou Dezhong, Greiser Udo, Guo Tianying, Guo Rui, Wang Wenxin
Charles Institute of Dermatology, School of Medicine, University College Dublin, Belfield, Dublin 4, Ireland.
Key Laboratory of Functional Polymer Materials, Ministry of Education, Institute of Polymer Chemistry, College of Chemistry, Nankai University, 300071, Tianjin, China.
ACS Biomater Sci Eng. 2017 Jul 10;3(7):1283-1286. doi: 10.1021/acsbiomaterials.6b00503. Epub 2016 Oct 26.
To enhance the gene transfection efficiency to targeted cells while reducing the side effects to untargeted cells is of great significance for clinical gene therapy. Here, biodegradable highly branched poly(β-amino ester)s (HPAESS) are synthesized and functionalized with folate (HPAESS-FA) and lactobionic acid (HPAESS-Lac) for targeted cancer cell gene transfection. Results show that because of the triggered degradability of the vector and enhanced receptor-mediated cellular uptake of polyplexes, the HPAESS-FA and HPAESS-Lac exhibit superior gene transfection capability in specific cancer cells with negligible cytotoxicity, pointing to their promise as targeted vectors for efficient cancer gene therapy.
提高基因转染到靶细胞的效率，同时降低对非靶细胞的副作用，对临床基因治疗具有重要意义。在此，合成了可生物降解的高度支化聚（β-氨基酯）（HPAESS），并用叶酸（HPAESS-FA）和乳糖酸（HPAESS-Lac）进行功能化，用于靶向癌细胞基因转染。结果表明，由于载体的触发降解性和增强的受体介导的多聚体细胞摄取，HPAESS-FA和HPAESS-Lac在特定癌细胞中表现出优异的基因转染能力，细胞毒性可忽略不计，表明它们有望成为高效癌症基因治疗的靶向载体。
