Gilleron Jerome, Paramasivam Prasath, Zeigerer Anja, Querbes William, Marsico Giovanni, Andree Cordula, Seifert Sarah, Amaya Pablo, Stöter Martin, Koteliansky Victor, Waldmann Herbert, Fitzgerald Kevin, Kalaidzidis Yannis, Akinc Akin, Maier Martin A, Manoharan Muthiah, Bickle Marc, Zerial Marino
Max Planck Institute of Molecular Cell Biology and Genetics, Pfotenhauerstrasse 108 01307, Dresden, Germany INSERM U1065, Centre Méditerranéen de Médecine Moléculaire C3M, Nice, France; Université de Nice Sophia-Antipolis, Nice, France.
Max Planck Institute of Molecular Cell Biology and Genetics, Pfotenhauerstrasse 108 01307, Dresden, Germany.
Nucleic Acids Res. 2015 Sep 18;43(16):7984-8001. doi: 10.1093/nar/gkv762. Epub 2015 Jul 28.
Most delivery systems for small interfering RNA therapeutics depend on endocytosis and release from endo-lysosomal compartments. One approach to improve delivery is to identify small molecules enhancing these steps. It is unclear to what extent such enhancers can be universally applied to different delivery systems and cell types. Here, we performed a compound library screen on two well-established siRNA delivery systems, lipid nanoparticles and cholesterol conjugated-siRNAs. We identified fifty-one enhancers improving gene silencing 2-5 fold. Strikingly, most enhancers displayed specificity for one delivery system only. By a combination of quantitative fluorescence and electron microscopy we found that the enhancers substantially differed in their mechanism of action, increasing either endocytic uptake or release of siRNAs from endosomes. Furthermore, they acted either on the delivery system itself or the cell, by modulating the endocytic system via distinct mechanisms. Interestingly, several compounds displayed activity on different cell types. As proof of principle, we showed that one compound enhanced siRNA delivery in primary endothelial cells in vitro and in the endocardium in the mouse heart. This study suggests that a pharmacological approach can improve the delivery of siRNAs in a system-specific fashion, by exploiting distinct mechanisms and acting upon multiple cell types.
大多数用于小干扰RNA治疗的递送系统依赖于内吞作用以及从内溶酶体区室释放。一种改善递送的方法是鉴定增强这些步骤的小分子。目前尚不清楚此类增强剂在何种程度上可普遍应用于不同的递送系统和细胞类型。在此,我们对两种成熟的siRNA递送系统——脂质纳米颗粒和胆固醇缀合的siRNAs进行了化合物文库筛选。我们鉴定出51种增强剂,可使基因沉默提高2至5倍。令人惊讶的是,大多数增强剂仅对一种递送系统具有特异性。通过定量荧光和电子显微镜相结合的方法,我们发现这些增强剂的作用机制存在显著差异,它们要么增加内吞摄取,要么增加siRNAs从内体的释放。此外,它们通过不同机制调节内吞系统,作用于递送系统本身或细胞。有趣的是,几种化合物在不同细胞类型上均显示出活性。作为原理验证,我们表明一种化合物在体外原代内皮细胞以及小鼠心脏的心内膜中均可增强siRNA递送。这项研究表明,药理学方法可通过利用不同机制并作用于多种细胞类型,以系统特异性的方式改善siRNAs的递送。