Structure-Activity Relationship of Polyester-Based Cationic Polyrotaxane Vector-Mediated In Vitro siRNA Delivery: Effect on Gene Silencing Efficiency.

A series of linear polyester-based, α-cyclodextrin (CD)-threaded polyrotaxanes (PRTx) were synthesized for siRNA delivery. The investigation into the effect of the presence of polyester linkages in polyrotaxane established the structural-activity relationship between polyrotaxane and siRNA transfection efficiency. The ester-based polyrotaxane exhibited higher threading efficiency than poly(ethylene glycol)-based polyrotaxane.

RNA interference-based therapeutics: molecular platforms for infectious diseases.

The potential uses and therapeutic benefits of RNA interference (RNAi) are enormous. Recent insights into RNAi technologies have highlighted their role in analyzing the functions and regulation of gene expression in eukaryotes and further utilizing this information for identification and amelioration of many diseases. These studies have also established the role of RNAi mediated post-transcriptional gene silencing (PTGS) mechanism in mammals by several endogenous, gene regulation systems including small interfering RNAs (siRNA), micro RNA (miRNA) and small hairpin RNAs (shRNA).