Amino acids functionalized dendrimers with nucleus accumulation for efficient gene delivery
Amino acids functionalized dendrimers with nucleus accumulation for efficient gene delivery
复制标题
氨基酸功能化树枝状聚合物，具有核积累功能，可实现有效的基因递送
DOI：
10.1016/j.ijpharm.2021.120641
复制
发表时间：
2021
影响因子：
5.8
通讯作者：
Pan Weisan
中科院分区：
文献类型：
--
作者：
Tan Guoxin;Li Jiayang;Liu D;an;Pan Hao;Zhu Renfang;Yang Yue;Pan Weisan
Gene therapy is a promising approach to many diseases, however, the barriers in the gene delivery restrict its application. Therefore, in the present study, an efficient non-viral gene vector (PRHF/N/D) for overcoming the barriers in gene delivery was prepared. The synthesized PRHF integrated the advantages of PAMAM and amino acids, which could improve the cellular uptake, enhance the endosomal escape ability and minimize cytotoxicity. To further enhance nuclear entry of carrier, the nuclear localization signal (NLS) peptide was selected to add in the PRHF/D polyplexes. The PRHF/N/D polyplexes demonstrated good condensation capacity, wonderful pDNA protection and low toxicity. Moreover, the PRHF/N/D polyplexes showed the excellent transfection efficiency than P/D. PRHF/N/D further improve transfection capability than PRHF/D in the presence of NLS. After 4 h of incubation, the mean fluorescence intensity of PRHF/N/D was also higher than the P/D and PRHF/D complexes. We then investigated the intracellular dissociation, the DNA is able to disassemble from PRHF/N/D gene carriers. Taken together, we exhibited that this PRHF/N/D polyplexes has the potential for use in the gene delivery.
