Description:

Background

Small interfering RNA (siRNA) is a powerful tool to control cellular processes at a post-transcriptional level. It is a short double-strand RNA that can silence the specific gene expression by triggering the cleavage of a target messenger RNA (mRNA) at the post-transcriptional level in the cytoplasm. Its great potency is due to the high sequence-specific inhibition efficiency. Thus, the strategy of introducing the siRNA has been considered strongly for the down-regulation of certain proteins in the areas of functional genomics and genomic therapeutics. However, therapeutic applications of this strategy are still limited because of the instability of siRNA against nucleases. 

This invention presents a way to enhance the stability of siRNA. 

Technology Overview

In this invention, a double-strand siRNA can be modified with a phospholipid via a functional group linkage to increase its stability against nucleases and to facilitate siRNA liberation. These siRNA conjugates are useful for an application requiring the control of over-expressed genes mediating disease states. The chemical conjugation and the “lipidic” modification on the siRNA make it valuable because of its application in some lipid-based drug delivery systems (such as micelles and liposomes) in which the lipidic tail can be incorporated into the lipidic core of these biodevices (improving the protection and the delivery the siRNA) to reduce or inhibit the expression of a target polypeptide, e.g., to treat a disease or a disorder. 

Benefits

  • Stabilization and delivery of small interfering RNA (siRNA) and enhanced stability against nucleases 
  • Reversible binding in-vivo, making siRNA free to act on the target moiety 
  • Enhanced solubility 
  • Commercially viable for knock-down of undesirable genes for related therapeutic effects 

Applications

  • Global RNAi drug delivery 
  • Global RNAi nucleic acid drug delivery 

Opportunity

  • License
  • Partnering
  • Research collaboration
Patent Information:
For Information, Contact:
Mark Saulich
Associate Director of Commercialization
Northeastern University
m.saulich@northeastern.edu
Inventors:
Vladimir Torchilin
Tiziana Musacchio
Keywords: