A High-Throughput Platform for Formulating and Screening Multifunctional Nanoparticles Capable of Simultaneous Delivery of Genes and Transcription Factors.
A High-Throughput Platform for Formulating and Screening Multifunctional Nanoparticles Capable of Simultaneous Delivery of Genes and Transcription Factors.
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DOI：
10.1002/anie.201507546
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发表时间：
2016-01-04
期刊：
影响因子：
--
通讯作者：
Wang H
中科院分区：
文献类型：
--
作者：
Liu Y;Du J;Choi JS;Chen KJ;Hou S;Yan M;Lin WY;Chen KS;Ro T;Lipshutz GS;Wu L;Shi L;Lu Y;Tseng HR;Wang H
同时递送多种基因和蛋白质（例如，转录因子（TF）由于其调节细胞回路的能力而成为围绕治疗研究的新兴问题。然而，目前的基因和蛋白质递送策略是基于缓慢的分批合成，其是无效的、控制不良的，并且不能同时递送具有协同功能的基因和蛋白质。因此，该领域的进展仅限于体外研究。在这里，通过将微流控技术与超分子合成策略相结合，我们提出了一种高通量的方法，用于配制和筛选多功能超分子纳米颗粒（MFSNPs），这些纳米颗粒从功能模块的集合中自组装，以实现在体外和体内以前所未有的效率同时递送一个基因和TF。我们设想这种新方法可以为免疫治疗、干细胞重编程和其他治疗应用开辟一条新途径。
Simultaneous delivery of multiple genes and proteins (e.g., transcription factors, TFs) is an emerging issue surrounding therapeutic research due to their ability to regulate cellular circuitry. Current gene and protein delivery strategies, however, are based on slow batch synthesis, which is ineffective, poorly controlled, and incapable of simultaneous delivery of both genes and proteins with synergistic functions. Consequently, advances in this field have been limited to in vitro studies. Here, by integrating microfluidic technologies with a supramolecular synthetic strategy, we present a high-throughput approach for formulating and screening multifunctional supramolecular nanoparticles (MFSNPs) self-assembled from a collection of functional modules to achieve simultaneous delivery of one gene and TF with unprecedented efficiency both in vitro and in vivo. We envision that this new approach could open a new avenue for immunotherapy, stem cell reprogramming, and other therapeutic applications.
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