Pluronic-based nanoparticles as novel DNA transfection vectors

基于 Pluronic 的纳米粒子作为新型 DNA 转染载体

• 批准号: 355626-2011
• 负责人: Wettig, Shawn
• 金额: $ 1.53万
• 依托单位: University of Waterloo
• 依托单位国家: 加拿大
• 项目类别: Discovery Grants Program - Individual
• 财政年份: 2013
• 资助国家: 加拿大
• 起止时间: 2013-01-01 至 2014-12-31
• 项目状态: 已结题
• 来源: https://www.nserc-crsng.gc.ca/ase-oro/Details-Detailles_eng.asp?id=527842
• 关键词: Pluronic; based; nanoparticles; novel; DNA

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.

基因疗法作为治疗人类健康中的任何数量的疾病（遗传和/或自然获得的）具有巨大的潜力，其中可以将缺陷或缺失基因的正确拷贝递送到细胞以恢复正常功能。然而，为了实现这一潜力，必须在用于递送遗传信息的药物递送系统方面取得重大进展。 目前，最有效的递送系统是基于病毒，并利用病毒感染细胞的固有能力，递送DNA。 不幸的是，围绕基于病毒的系统的使用存在非常真实的安全性问题，这些系统可能导致突然和严重的免疫应答，这些免疫应答在临床试验中与患者死亡有关。