Márquez-Miranda Valeria, Peñaloza Juan Pablo, Araya-Durán Ingrid, Reyes Rodrigo, Vidaurre Soledad, Romero Valentina, Fuentes Juan, Céric Francisco, Velásquez Luis, González-Nilo Fernando D, Otero Carolina
Facultad de Biología, Center for Bioinformatics and Integrative Biology (CBIB), Universidad Andres Bello, Republica 239, Santiago, Chile.
Fundación Fraunhofer Chile Research, M. Sánchez Fontecilla 310 piso 14, Las Condes, Chile.
Nanoscale Res Lett. 2016 Dec;11(1):66. doi: 10.1186/s11671-016-1260-9. Epub 2016 Feb 4.
Poly(amidoamine) dendrimers are the most recognized class of dendrimer. Amino-terminated (PAMAM-NH2) and hydroxyl-terminated (PAMAM-OH) dendrimers of generation 4 are widely used, since they are commercially available. Both have different properties, mainly based on their different overall charges at physiological pH. Currently, an important function of dendrimers as carriers of short single-stranded DNA has been applied. These molecules, known as antisense oligonucleotides (asODNs), are able to inhibit the expression of a target mRNA. Whereas PAMAM-NH2 dendrimers have shown to be able to transfect plasmid DNA, PAMAM-OH dendrimers have not shown the same successful results. However, little is known about their interaction with shorter and more flexible molecules such as asODNs. Due to several initiatives, the use of these neutral dendrimers as a scaffold to introduce other functional groups has been proposed. Because of its low cytotoxicity, it is relevant to understand the molecular phenomena involving these types of dendrimers. In this work, we studied the behavior of an antisense oligonucleotide in presence of both types of dendrimers using molecular dynamics simulations, in order to elucidate if they are able to form stable complexes. In this manner, we demonstrated at atomic level that PAMAM-NH2, unlike PAMAM-OH, could form a well-compacted complex with asODN, albeit PAMAM-OH can also establish stable interactions with the oligonucleotide. The biological activity of asODN in complex with PAMAM-NH2 dendrimer was also shown. Finally, we revealed that in contact with PAMAM-OH, asODN remains outside the cells as TIRF microscopy results showed, due to its poor interaction with this dendrimer and cell membranes.
聚(酰胺胺)树枝状大分子是最受认可的一类树枝状大分子。第四代氨基端基(PAMAM-NH2)和羟基端基(PAMAM-OH)树枝状大分子被广泛使用,因为它们有商业产品。两者具有不同的性质,主要基于它们在生理pH值下不同的整体电荷。目前,树枝状大分子作为短单链DNA载体的一项重要功能已得到应用。这些分子被称为反义寡核苷酸(asODNs),能够抑制靶mRNA的表达。虽然PAMAM-NH2树枝状大分子已显示能够转染质粒DNA,但PAMAM-OH树枝状大分子并未显示出同样成功的结果。然而,对于它们与更短且更灵活的分子(如asODNs)之间的相互作用却知之甚少。由于多项举措,有人提出使用这些中性树枝状大分子作为支架来引入其他官能团。因其低细胞毒性,了解涉及这类树枝状大分子的分子现象很重要。在这项工作中,我们使用分子动力学模拟研究了反义寡核苷酸在两种树枝状大分子存在下的行为,以阐明它们是否能够形成稳定的复合物。通过这种方式,我们在原子水平上证明,与PAMAM-OH不同,PAMAM-NH2可以与asODN形成紧密的复合物,尽管PAMAM-OH也能与寡核苷酸建立稳定的相互作用。还展示了与PAMAM-NH2树枝状大分子复合的asODN的生物活性。最后,我们发现,如全内反射荧光显微镜(TIRF)显微镜结果所示,与PAMAM-OH接触时,asODN留在细胞外,这是因为它与这种树枝状大分子和细胞膜的相互作用较弱。