Synthesis of Amphiphilic Poly(β-amino ester) for Efficiently Minicircle DNA Delivery in Vivo
Synthesis of Amphiphilic Poly(β-amino ester) for Efficiently Minicircle DNA Delivery in Vivo
复制标题
两亲性聚（β-氨基酯）的合成，用于体内有效的小环 DNA 传递
DOI：
10.1021/acsami.6b04412
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发表时间：
2016
影响因子：
9.5
通讯作者：
Chen Zhi-Ying
中科院分区：
文献类型：
--
作者：
Zhao Jing;Huang Ping;Wang Zhiyong;Tan Yan;Hou Xiaohu;Zhang Liping;He Cheng-Yi;Chen Zhi-Ying
微环DNA（minicircleDNA，mcDNA）是一种增强型非病毒DNA载体，具有良好的生物安全性和转基因表达特性。本文中，我们报道了一种新型的两亲性聚合物，其包含聚乙烯亚胺（PEI）修饰的聚（β-氨基酯）PEI-PBAE（C16），用于有效的mcDNA体内递送。合成的聚合物可以将mcDNA浓缩成纳米级结构，并表现出有效的基因转染能力，而没有可检测到的细胞毒性。重要的是，当腹膜内注射到小鼠中时，这些PEI-PBAE（C16）纳米复合物能够导致持续至少72小时的高水平的转基因表达。总体而言，这些结果表明PEI-PBAE（C16）可以介导体内有效且安全的基因递送，具有临床应用潜力。
Minicircle DNA (mcDNA) is a kind of enhanced nonviral DNA vector with excellent profiles in biosafety and transgene expression. Herein, we reported a novel amphiphilic polymer comprising polyethylenimine(PEI) modified Poly(β-amino ester) PEI–PBAE(C16) for efficient mcDNA delivery in vivo. The synthesized polymer could condense mcDNA into nanoscaled structure and exhibited efficient gene transfection ability without detectable cytotoxicity. Importantly, when injected into mouse intraperitoneally, these PEI–PBAE(C16) nanocomplexes were able to result in high level of trangene expression which lasted at least 72 h. Overall, these results demonstrated the PEI–PBAE(C16) can mediate effective and safe gene delivery in vivo with clinical application potential.