Efficient Cytosolic Delivery of siRNA Using Nanoparticle-Stabilized Nanocapsules

PRODUCT OPPORTUNITIES

•       Novel delivery vehicle for the development and delivery of RNAi-based therapeutics

•       Advanced tool for life science, drug discovery or biomedical research

•       Efficient intracellular nucleic acid delivery system

 

 

ADVANTAGES

•       New mechanism of siRNA delivery that allows for efficient and rapid cytosolic delivery of siRNA

•       No endosomal entrapment of siRNA

•       Stable nanocapsules

•       High-efficiency gene silencing

•       Applicable to the cytosolic delivery of other nucleic acids

 

 

 

TECHNOLOGY DESCRIPTION

This invention provides a new siRNA delivery vehicle that enables efficient cytosolic delivery of siRNA as well as other nucleic acids. The delivery system involves the use of surface functionalized nanoparticles to form self-assembled superstructures with the protein to be delivered. The nanoparticle-protein assemblies effectively escape endosomal entrapment and rapidly deliver the protein into the cell cytosol or the targeting organelle. This protein delivery system has been successfully demonstrated for the efficient delivery of the CRISPR/Cas9 gene editing system as well as a number of other proteins with different physiochemical properties.

 

Nanocapsule-based siRNA delivery resulted in efficient knockdown of PLK1 protein in cancer cells

 

 

ABOUT THE LEAD INVENTOR

Dr. Vincent M. Rotello is the Charles A. Goessmann Professor of Chemistry at the University of Massachusetts Amherst. His research team focuses on creating new functional materials for a variety of applications.

 

 

AVAILABILITY:

Available for Licensing and/or Sponsored Research

 

 

DOCKET:

UMA 15-057

 

 

PATENT STATUS:

Patent Pending

 

 

NON-CONFIDENTIAL INVENTION DISCLOSURE

 

 LEAD INVENTOR:

Vincent M. Rotello, Ph.D.

 

 

CONTACT:

 

This invention provides a general and efficient protein delivery platform that enables intracellular delivery of proteins having different physiochemical properties. The delivery system involves the use of surface functionalized nanoparticles to form self-assembled superstructures with the protein to be delivered. The nanoparticle-protein assemblies effectively escape endosomal entrapment and rapidly deliver the protein into the cell cytosol or the targeting organelle. This protein delivery system has been successfully demonstrated for the efficient delivery of the CRISPR/Cas9 gene editing system as well as a number of other proteins with different physiochemical properties.

Patent Information: