Defined pH-sensitive nanogels as gene delivery platform for siRNA mediated in vitro gene silencing,Biomaterials Science

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Defined pH-sensitive nanogels as gene delivery platform for siRNA mediated in vitro gene silencing
Biomaterials Science ( IF 5.8 ) Pub Date : 2017-10-16 00:00:00 , DOI: 10.1039/c7bm00729a
Mathias Dimde _{1,

2,

3,

4} , Falko Neumann _{1,

2,

3,

4} , Felix Reisbeck _{1,

2,

3,

4} , Svenja Ehrmann _{1,

2,

3,

4,

5} , Jose Luis Cuellar-Camacho _{1,

2,

3,

4} , Dirk Steinhilber _{1,

2,

3,

4} , Nan Ma _{1,

2,

3,

4,

6} , Rainer Haag _{1,

2,

3,

4}

Affiliation

In the present study, a pH sensitive nanogel platform for gene delivery was developed. The cationic nanogels based on dendritic polyglycerol (dPG) and low molecular weight polyethylenimine units were able to encapsulate siRNA during the manufacturing process. The thiol-Michael nanoprecipitation method, which operates under mild conditions and did not require any catalyst or surfactant, was used to develop tailor-made nanogels in the sub-100 nm range. The incorporation of pH sensitive benzacetal-bonds inside the nanogel network enables the controlled intracellular release of the cargo. The functionality to transport therapeutic biomolecules was tested by an in vitro GFP-siRNA transfection assay. Encapsulated siRNA could silence GFP expressing HeLa cells (up to 71% silencing in GFP). Furthermore, significantly reduced toxicity of the nanogel platform compared to the non-degradable PEI was observed. These properties realize a new carrier platform in the field of gene therapy.

中文翻译：

定义pH敏感的纳米凝胶作为siRNA介导的体外基因沉默的基因传递平台

在本研究中，开发了用于基因传递的pH敏感纳米凝胶平台。基于树突状聚甘油（dPG）和低分子量聚乙烯亚胺单元的阳离子纳米凝胶能够在制造过程中包裹siRNA。硫醇-迈克尔纳米沉淀法在温和的条件下运行，不需要任何催化剂或表面活性剂，用于开发低于100 nm范围的量身定制的纳米凝胶。在纳米凝胶网络内部并入pH敏感的苯乙缩醛键可实现货物在细胞内的受控释放。体外测试了治疗性生物分子的运输功能GFP-siRNA转染测定。封装的siRNA可以使表达GFP的HeLa细胞沉默（在GFP中沉默高达71％）。此外，观察到与不可降解的PEI相比，纳米凝胶平台的毒性显着降低。这些特性实现了基因治疗领域的新载体平台。

更新日期：2017-10-16

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