Engineering RNA for Targeted siRNA Delivery and Medical Application

Abstract

RNA engineering for nanotechnology and medical applications is an exciting emerging research field. RNA has intrinsically defined features on the nanometer scale and is a particularly interesting candidate for such applications due to its amazing diversity, flexibility and versatility in structure and function. Specifically, the current use of siRNA to silence target genes involved in disease has generated much excitement in the scientific community. The intrinsic ability to sequence-specifically down-regulate gene expression in a temporally-and spatially-controlled fashion has led to heightened interest and rapid development of siRNA-based therapeutics. Though methods for gene silencing with high efficacy and specificity have been achieved in vitro, the effective delivery of nucleic acids to specific cells in vivo has been a hurdle for RNA therapeutics. This review covers different RNA-based approaches for diagnosis, prevention and treatment of human disease, with a focus on the latest developments of nonviral carriers of siRNA for delivery in vivo. The applications and challenges of siRNA therapy, as well as potential solutions to these problems, the approaches for using phi29 pRNA-based vectors as polyvalent vehicles for specific delivery of siRNA, ribozymes, drugs or other therapeutic agents to specific cells for therapy will also be addressed.

Self-assemble gene delivery system for molecular targeting using nucleic acid aptamer

“…Aptamers are a family of RNA-or DNA-based oligonucleotides 20-50 nt in length that can specifically bind to selected targets (Guo, et al, 2010). They have often been described as nucleic acid versions of antibodies.…”

microRNA-186 inhibits cell proliferation and induces apoptosis in human esophageal squamous cell carcinoma by targeting SKP2

“…Oligonucleotide therapies using microRNA (miRNA), small interfering RNA (siRNA), or ribozyme have attracted particular attention because of their specificity and safety. [2][3][4] miRNAs, a class of small non-coding single-stranded RNAs that are 20-22 nucleotides long, are involved in multiple cellular biological processes, such as differentiation, proliferation, apoptosis, and migration by targeting the 3′ untranslated region (UTR) of mRNAs of target genes in a sequence-specific manner. [5][6][7] It has also been shown that miRNAs have a crucial role in carcinogenesis and cancer progression.…”

Drug delivery systems based on nucleic acid nanostructures

“…The most obvious use of oligonucleotide nanostructures in the medical field is the delivery of siRNA, antisense RNA or genes. However, several excellent reviews on this topic have been published recently [44][45][46][47]. The same holds true for DNA nanoobjects employed in the context of cellular and in-vivo bioimaging [48,49].…”