CRISPR Gene Correction: A New Horizon for Medical Care

We are in the midst of a revolution in genome engineering and CRISPR-Cas9 technology was the spark. With unprecedented rapidity, this technology has provided a straightforward, robust, and specific method for genome engineering and gene correction. CRISPR-Cas9 is a technique that allows for highly rapid modification of DNA in genomes of organisms and/or of cells. Our research focuses on developing CRISPR genome engineering as curative therapy for genetic diseases. Our lab is particularly interested in applying genome engineering for gene therapy of genetic disorders of the blood and the immune system such as severe combined immunodeficiency (SCID) also commonly known as the “bubble boy” disease. SCIDs are a set of life threatening genetic diseases in which patients are born with mutations in single genes, and are unable to develop functional immune system. We believe that the ultimate cure for these diseases will be transplantation of gene-corrected stem cells that create normal and healthy immune system. To be able to apply this approach in the clinic, we must assure that the CRISPR genome engineering technology is efficient and safe. Hence, our research focuses on developing an optimized CRISPR genome engineering for robust, specific and non-toxic gene correction. In my talk I will present the concept of CRISPR genome engineering and its possible application in medicine. I will present some of our recent studies demonstrating enhanced CRISPR genome editing by using chemical alterations to the CRISPR system, enabling therapeutically relevant genome editing frequencies in stem cells of the blood and the immune system. Finally, I will also present our joint research with the Yakhini group on developing an approach to accurately measure CRISPR specificity. Overall, we believe that by advancing CRISPR technology our research will accelerate the bench to bedside path and will help in generating a clear trajectory towards future clinical trials.