New Amphiphilic Amino Acid Derivatives for Efficient DNA Transfection in Vitro  

作　　者：Lucía C.Pena María F.Argaraná María M.De Zan Antonella Giorello Sebastián Antuna Claudio C.Prieto Carolina M.I.Veaute Diana M.Müller 

机构地区：[1]LAQUIMAP,Dto.Química Orgánica,Universidad Nacional del Litoral,Santa Fe,Argentina [2]Cát.Microbiología General,Universidad Nacional del Litoral,Santa Fe,Argentina [3]Laoratorio de Control de Medicamentos,Universidad Nacional del Litoral,Santa Fe,Argentina [4]Instituto de Investigaciones en Catálisis y Petroquímica,Universidad Nacional del Litoral,Santa Fe,Argentina [5]Laboratorio de Cultivos Celulares,Universidad Nacional del Litoral,Santa Fe,Argentina [6]Laboratorio de Inmunología Básica,Universidad Nacional del Litoral,Santa Fe,Argentina

出　　处：《Advances in Chemical Engineering and Science》2017年第2期191-205,共15页化学工程与科学期刊（英文）

基　　金：supported by grants from Universidad Nacional del Litoral(U.N.L)(CAI+D,2011),República Argentina.

摘　　要：Nucleic acids-based therapies have recently developed as next-generation agents for treating and preventing viral infection, cancer, and genetic disorders, but their use is still limited due to its relatively poor delivery into targeted cells. We designed and synthesized new amphiphilic amino acid derivatives (cysteine-based) of low molecular weight, formed by the same pentapeptide (AG2: WWCOO) N-acylated, with different hydrophobic chains containing from 12 to 18 carbons, named AG2-Cn (N), which dimerize by oxidation in the presence of pLenti-CMV-GFP Puro plasmid (P) in the respective gemini. We determined transfection efficiency, critical micelle concentration, particle size, ζ-potential and cytotoxicity for the derivatives obtained. We found that all the synthesized compounds were active for DNA delivery and had greater ability to transfect CHO-K1 cells. In particular, AG2-C18 is a promising carrier for gene delivery because it showed no cytotoxicity and its activity was greater than or equal to the commercial actives currently used.Nucleic acids-based therapies have recently developed as next-generation agents for treating and preventing viral infection, cancer, and genetic disorders, but their use is still limited due to its relatively poor delivery into targeted cells. We designed and synthesized new amphiphilic amino acid derivatives (cysteine-based) of low molecular weight, formed by the same pentapeptide (AG2: WWCOO) N-acylated, with different hydrophobic chains containing from 12 to 18 carbons, named AG2-Cn (N), which dimerize by oxidation in the presence of pLenti-CMV-GFP Puro plasmid (P) in the respective gemini. We determined transfection efficiency, critical micelle concentration, particle size, ζ-potential and cytotoxicity for the derivatives obtained. We found that all the synthesized compounds were active for DNA delivery and had greater ability to transfect CHO-K1 cells. In particular, AG2-C18 is a promising carrier for gene delivery because it showed no cytotoxicity and its activity was greater than or equal to the commercial actives currently used.

关 键 词：AMPHIPHILE N-Acylated CYSTEINE GEMINI ORNITHINE TRANSFECTION 

分 类 号：R73[医药卫生—肿瘤]