초록 |
To efficiently deliver plasmid DNA, we prepared bioreducible poly(amido amine)s by reacting cystamine bisacrylamide (CBA) with 34 different small amines. After screening, we selected lead substances and characterized. CBA-106 polymer had high serum stability and bioreducible ability under physiological conditions and showed relatively low cytotoxicity. To deliver mRNA for in vivo applications, we synthesized lipid-like materials, called the lipidoids using 8 of small amines and 4 of epoxide lipids. After screening, we selected lead lipidoids molecules, 102-C10. Our lead compound, 102-C10, was mixed with other liposome formulating components to efficiently deliver mRNA in vivo. Formulation showed that mRNA can be well delivered to the target tissue and protein expression can be achieved in vivo. From our results, we can conclude that the lead substances such as CBA-106 and 102-C10 have great potential as a novel delivery carrier for gene therapy applications. |