Leveraging DNA Repair to Enhance CRISPR Genome Editing

Type of Award: catalyst
Award Number: C-090
Award Period: March 2019 - February 2021
Amount Awarded: $ 250,000.00
PI(s): Leslyn Hanakahi, UIC; Stephen Kron, UChicago;

Abstract: CRISPR has raised hopes for treatment of most genetic disease. For many patients, simply correcting the mutation would be sufficient to dramatically improve function. Blood diseases have already been corrected in the laboratory and treated cells returned to the patient. However, for many genetic diseases that affect major organs, it may be necessary to correct mutations in patients. Currently, CRISPR remains far too inefficient. Though accurate in making a break in the target gene's DNA, only a minor fraction of cells are corrected, leaving the majority unchanged or with damaged genes. By leveraging their expertise in cellular responses to DNA damage, PI's Hanakahi at UIC and Kron at UC hope to dramatically increase the efficiency of CRISPR. They will focus on changing how cells respond to CRISPR breaks to promote gene correction, using approaches that can be safely translated to patients, treating genetic diseases before damage is irreversible.