Efficient Site-Specific Integration Of New Genetic Information Into Human Cells

Tech ID: 24215 / UC Case 2014-199-0

Patent Status

Country Type Number Dated Case
European Patent Office Published Application 3188763 07/12/2017 2014-199
Patent Cooperation Treaty Published Application WO2016036754 03/10/2016 2014-199
 

Additional Patent Pending

Brief Description


 

The CRISPR/Cas9 system is a robust genome editing technology that works based on the RNA-programmed DNA cleaving activity of the Cas9 enzyme.  Cas9 generates blunt double-strand DNA (dsDNA) breaks (DSBs) at sites defined by a guide sequence contained within an associated CRISPR RNA (crRNA) transcript and has transformed the ability to engineer eukaryotic organisms by initiating DNA repair pathways that lead to targeted genetic re-programming.  There is a need, however, for methods that increase the efficiency of target DNA modification by Cas9 targeting complexes and/or increase the effectiveness of such methods.

 

UC Berkeley researchers have found a simple and robust approach for introducing new genetic information site-specifically, in a eukaryotic cell, and with high efficiency using timed delivery of Cas9-guide RNA ribonucleoprotein (RNP) complexes.  The approach combines well-established cell cycle synchronization techniques with direct nucleofection of the pre-assembled Cas9 ribonucleoprotein (RNP) complexes to achieve controlled nuclease action at the phase of the cell cycle best for HDR.

 

Suggested uses


  • Genome editing (e.g., human, plants, and animal cells)
  • Genome engineering in both transformed and primary human cells

Advantages


  • Enhanced HDR efficiency in human cells
  • Highly efficient, site-specific editing
  • Simple and robust editing
  • Cell mortality minimized

Publication

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Inventors

  • Doudna, Jennifer A.

Other Information

Keywords

Genetic engineering, homology-directed repair, HDR, genome editing, Cas9, CRISPR

Categorized As