CRISPR-SKIP System to Modulate Expression of Exons & Improve Cellular Delivery of DNA Editing Systems

Dr. Perez-Pinera has developed a new way to edit genes using CRISPR technology that promises to be safer and more efficient that existing technologies. His method uses single base editing, to avoid double-stranded breaks in DNA and induce a mutation that causes exon skipping to occur which can silence, knockdown or ameliorate the effects of downstream gene products. Avoiding double stranded breaks eliminates the possibility of malignant mutations which can result from improper non-homologous end joining. He also demonstrates a novel delivery method for the CRISPR-cas9 system using a split base editor inserted into an adeno-associated virus to realize greater, more efficacious delivery into the cell. This method can be used to treat diseases such as Duchenne’s Muscular Dystrophy with more permanence than current treatments as well as Huntington’s and cardiovascular diseases.