US EPA

4239649 

Journal Article 

Biocompatible fluorinated poly(β-amino ester)s for safe and efficient gene therapy 

Gong, JH; Wang, Y; Xing, L; Cui, PF; Qiao, JB; He, YJ; Jiang, HL 

2018 

Yes 

International Journal of Pharmaceutics
ISSN: 0378-5173
EISSN: 1873-3476 

535 

1-2 

180-193 

English 

29129572 

10.1016/j.ijpharm.2017.11.015 

Cationic polymers have been widely used as one of the most promising non-viral vehicles for gene delivery due to their potential safety and ease of large-scale production. Here, we report the design and synthesis of a series of novel biodegradable fluorinated poly(β-amino ester)s (FPBAEs) by simple Michael-addition reaction as safe and efficient gene carrier. The results of transfection efficacy assay demonstrated the optimal FPBAE could mediated much higher GFP expression than the commercial transfection agents, polyethyleneimine (PEI, Mw = 25K) and Lipo 2000, as well as the non-fluorinated poly(β-amino ester)s (PBAE) on both HeLa and HEK-293T cell lines (higher than 70 and 90%, respectively), which was largely attributed to fluorination. Moreover, MTT and hemolysis assay indicated a preferable biocompatibility of FPBAE compared with PEI 25K owing to the low molecular weight and the presence of cleavable ester bonds. Taken together, the novel polymer FPBAE with both excellent gene transfection efficacy and much lower toxicity could serve as a desirable gene vector.