Small Molecule Assisted Cell Penetrating Cas9 RNP Delivery

Tech ID: 27354 / UC Case 2017-098-0

Patent Status

Country Type Number Dated Case
Patent Cooperation Treaty Published Application WO2019036185 02/21/2019 2017-098
 

Additional Patent Pending

Brief Description


Clustered regularly interspaced short palindromic repeats (CRISPR) Cas systems provide a means for modifying genomic information and have the potential to revolutionize the treatment of genetic diseases. Although RNA-programmed Cas9 has proven to be a versatile tool for genome engineering in multiple cell types and organisms, it has been challenging to develop the therapeutics because they require the simultaneous in vivo delivery of the Cas9 protein, guide RNA and donor DNA. Compositions that can increase the efficiency of such delivery, particular in eukaryotic cells, are greatly needed.

 

UC Researchers have discovered that the inclusion of an agent that decreases the acidity of an endosome inside eukaryotic cells, in a genome editing composition, increased the efficiency of genome editing.  The agent was included in a composition having an RNA-guided endonuclease and an RNA-guided endonuclease and was used for gene editing.

Suggested uses


  • Genome editing (particularly in eukaryotic cells)
  • Research reagent
  • Gene therapy
  • Increased Delivery of CRISPR Cas components to eukaryotic cells

Advantages

  • Significantly increased gene editing efficiency through increased delivery through endosome

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Inventors

  • Doudna, Jennifer A.

Other Information

Keywords

CRISPR, delivery, eukaryotic cells

Categorized As