作者
Bingyang Shi, Meng Zheng, Wei Tao, Roger Chung, Dayong Jin, Dariush Ghaffari, Omid C Farokhzad
发表日期
2017/8/14
来源
Biomacromolecules
卷号
18
期号
8
页码范围
2231-2246
出版商
American Chemical Society
简介
After more than 20 years of intensive investigations, gene therapy has become one of the most promising strategies for treating genetic diseases. However, the lack of ideal delivery systems has limited the clinical realization of gene therapy’s tremendous potential, especially for DNA-based gene therapy. Over the past decade, considerable advances have been made in the application of polymer-based DNA delivery systems for gene therapy, especially through multifunctional systems. The core concept behind multifunctional polymeric DNA delivery systems is to endow one single DNA carrier, via materials engineering and surface modification, with several active functions, e.g., good cargo DNA protection, excellent colloidal stability, high cellular uptake efficiency, efficient endo/lysosome escape, effective import into the nucleus, and DNA unpacking. Such specially developed vectors would be capable of …
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