Institute of Toxicology and Genetics, Karlsruhe Institute of Technology (KIT), Hermann-von-Helmholtz Platz 1, 76344, Eggenstein-Leopoldshafen, Germany.
University of Heidelberg, Department of Applied Physical Chemistry, 69120, Heidelberg, Germany.
Adv Mater. 2016 Feb 10;28(6):1159-75. doi: 10.1002/adma.201502888. Epub 2015 Nov 26.
Nanoparticles based on cationic polymers, lipids or lipidoids are of great interest in the field of gene delivery applications. The research on these nanosystems is rapidly growing as they hold promise to treat wide variety of human diseases ranging from viral infections to genetic disorders and cancer. Recently, combinatorial design principles have been adopted for rapid generation of large numbers of chemically diverse polymers and lipids capable of forming multifunctional nanocarriers for the use in gene delivery applications. At the same time, current high-throughput screening systems as well as convenient cell assays and readout techniques allow for fast evaluation of cell transfection efficiencies and toxicities of libraries of novel gene delivery agents. This allows for a rapid evaluation of structure-function relationship as well as identification of novel efficient nanocarriers for cell transfection and gene therapy. Here, the recent contribution of high-throughput synthesis to the development of novel nanocarriers for gene delivery applications is described.
基于阳离子聚合物、脂质或类脂的纳米颗粒在基因传递应用领域具有重要的研究意义。这些纳米系统的研究正在迅速发展，因为它们有望治疗从病毒感染到遗传疾病和癌症等多种人类疾病。最近，组合设计原则已被用于快速生成大量具有不同化学性质的聚合物和脂质，这些聚合物和脂质能够形成多功能纳米载体，用于基因传递应用。同时，目前的高通量筛选系统以及方便的细胞检测和读出技术允许快速评估新型基因传递剂文库的细胞转染效率和毒性。这使得能够快速评估结构-功能关系，并确定用于细胞转染和基因治疗的新型高效纳米载体。本文描述了高通量合成在新型基因传递应用纳米载体开发中的最新贡献。
