Advanced Therapeutic Platforms based on Non-Classical Tripodal Interfering RNA Structural Format

DOI: 10.14800/rd.708

Abstract

The flexibility of human RNAi (RNA interference) machinery to accommodate unconventional structures has opened the way for the development of novel RNAi triggering formats, suitable for advanced therapeutic applications. Non-classical siRNA structural variants show improved functional features over the conventional siRNA formats including increased potency, reduced non-specific responses, and enhanced cellular delivery. These non-classical RNAi structures with enhanced structural and functional properties, in combination with appropriate delivery vehicles, can be an effective therapeutic module. Here we summarize our recent efforts on expanding the structural repertoire of classical siRNAs, using tripodal interfering RNA formats, to develop novel RNAi based therapeutics for advanced health-care applications.