Xiao xuan LIU
China Pharmaceutical University, P. R. China
Title: Stimulus-sensitive self-assembling amphiphilic dendrimers as siRNA delivery platform
Biography
Biography: Xiao xuan LIU
Abstract
RNAi with synthetic siRNA holds great promise for therapeutic applications. However, safe and successful clinical translation essentially requires further advancement of developing efficient delivery systems. Among myriad nanocarriers, amphiphilic dendrimers, marrying the characteristic of dendrimers, self-assembly performance of amphiphilic molecules and the bio-mimicry of lipids, become particularly appealing as nanovectors for drug delivery in nanomedicine, in particular for small interfering RNA (siRNA) therapeutics. Here, we reported a series of amphiphilic dendrimers are able to self-assemble into adaptive supramolecular assemblies upon interaction with siRNA, and effectively delivers siRNAs to various cell lines, including human primary and stem cells, thereby outperforming the currently available nonviral vectors. Furthermore, a fluorinated bola-amphiphilic dendrimer was constructed for on-demand delivery of siRNA based on specific response to reactive oxygen species (ROS). The siRNA/dendrimer complexes disassemble efficiently in ROS-rich cancer cells, followed by the effective siRNA delivery and potent gene silencing. Thanks to the fluorine atoms, the ROS-responsive siRNA delivery process can be tracked through 19F-NMR analysis. Our study demonstrates that the self-assembling amphiphilic dendrimers represent novel and versatile means for functional siRNA delivery, heralding a new age of dendrimer-based self-assembled drug delivery in biomedical applications.