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A cationic amphiphilic co-polymer as a carrier of nucleic acid nanoparticles (Nanps) for controlled gene silencing, immunostimulation, and biodistribution.
Nanomedicine: Nanotechnology, Biology and Medicine ( IF 5.4 ) Pub Date : 2019-10-25 , DOI: 10.1016/j.nano.2019.102094
Justin R Halman 1 , Ki-Taek Kim 2 , So-Jung Gwak 2 , Richard Pace 2 , M Brittany Johnson 3 , Morgan R Chandler 1 , Lauren Rackley 1 , Mathias Viard 4 , Ian Marriott 3 , Jeoung Soo Lee 2 , Kirill A Afonin 1
Affiliation  

Programmable nucleic acid nanoparticles (NANPs) provide controlled coordination of therapeutic nucleic acids (TNAs) and other biological functionalities. Beyond multivalence, recent reports demonstrate that NANP technology can also elicit a specific immune response, adding another layer of customizability to this innovative approach. While the delivery of nucleic acids remains a challenge, new carriers are introduced and tested continuously. Polymeric platforms have proven to be efficient in shielding nucleic acid cargos from nuclease degradation while promoting their delivery and intracellular release. Here, we venture beyond the delivery of conventional TNAs and combine the stable cationic poly-(lactide-co-glycolide)-graft-polyethylenimine with functionalized NANPs. Furthermore, we compare several representative NANPs to assess how their overall structures influence their delivery with the same carrier. An extensive study of various formulations both in vitro and in vivo reveals differences in their immunostimulatory activity, gene silencing efficiency, and biodistribution, with fibrous NANPs advancing for TNA delivery.

中文翻译:

阳离子两亲共聚物,可作为核酸纳米颗粒(Nanps)的载体,用于控制基因沉默,免疫刺激和生物分布。

可编程核酸纳米颗粒(NANP)提供治疗性核酸(TNA)和其他生物学功能的受控配位。除了多价以外,最近的报道表明,NANP技术还可以引发特定的免疫反应,为这种创新方法增加了另一层可定制性。尽管核酸的传递仍然是一个挑战,但不断引入新的载体并对其进行测试。聚合平台已被证明可有效地保护核酸货物免遭核酸酶降解,同时促进其转运和细胞内释放。在这里,我们冒险超越了常规TNA的交付范围,并将稳定的阳离子聚(丙交酯-乙交酯)-接枝聚乙烯亚胺与功能化的NANP结合在一起。此外,我们比较了几个代表性的NANP,以评估它们的总体结构如何影响相同载体的递送。在体外和体内对各种制剂的广泛研究揭示了其免疫刺激活性,基因沉默效率和生物分布方面的差异,其中纤维性NANP促进TNA的递送。
更新日期:2019-10-25
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