An Efficient Intracellular Delivery Vehicle for the CRISPR/Cas9 Gene Editing System and Other Proteins
Vincent M. Rotello, Ph.D.
• Novel delivery vehicle for the development of revolutionary CRISPR/Cas9-based therapeutic methods to treat genetic diseases
• Advanced tool for life science, drug discovery or biomedical research
• Efficient intracellular protein delivery system
• A general and efficient delivery system applicable to proteins of different physiochemical properties
• Easier than gene-based expression
• Protein avoids endosomal entrapment during delivery
• Highly efficient nuclear delivery of the Cas9 protein
• Highly efficient delivery of a range of proteins into the cell cytosol or destination organelle
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.
Dr. Vincent M. Rotello is the Charles A. Goessmann Professor of Chemistry at the University of Massachusetts Amherst. His research group focuses on using nanoparticles as building blocks to create new functional materials for biological and biomedical applications.
Available for Licensing and/or Sponsored Research
UMA 15-046
Patent Issued
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