Pluronic-based nanoparticles as novel DNA transfection vectors

基因疗法具有巨大的潜力，可用于治疗人类健康中的许多疾病，包括遗传性和/或后天获得性疾病。基因疗法可将有缺陷或缺失基因的正确拷贝传送到细胞中，以恢复正常功能。然而，为了实现这一潜力，必须在用于传递遗传信息的药物传递系统方面取得重大进展。目前，最有效的传递系统是基于病毒，利用病毒感染细胞的固有能力，传递DNA。不幸的是，围绕使用基于病毒的系统存在非常现实的安全问题，这些系统可能导致突然和严重的免疫反应，在临床试验中与患者死亡有关。

Gene therapy has enormous potential as a treatment for any number of diseases in human health, both genetic and/or aquired in nature, where a correct copy of a defective or missing gene can be delivered to a cell to restore normal function. However, in order for this potential to be realized significant advancements must be made with respect to the drug delivery systems used to delivery genetic information. Currently, the most effective delivery systems are based upon viruses, and take advantage of the virus' inherent ability to infect cells, delivering DNA. Unfortunately there are very real saftey concerns surrounding the use of viral based systems, these systems can cause sudden and severe immune responses that have been linked to patient deaths in clinical trials.