Using CRISPR-Cas9 genome editing to enhance cell based therapies for the treatment of diabetes mellitus

© Springer International Publishing Switzerland 2016. Human pluripotent stem cells (hPSCs) have the ability to form all cell types of the body, making them an excellent potential source of insulin-producing pancreatic β-cells for diabetes treatment. To generate these cells in vitro requires a complete understanding of the normal process of pancreas development: an objective greatly aided by CRISPR-Cas9 genome-editing technology. First identified as the adaptive immune system of bacteria, CRISPR-Cas9 uses RNA to specifically target a DNA endonuclease to the genome, generating a double-strand break that can either be repaired by the error-prone NHEJ or via HDR. From the first demonstration that CRISPR-Cas can be programmed to cleave DNA in 2012, the field has advanced fast and now includes examples of targeting in many model organisms as well as gene knockout or reporter hPSC lines that will aid in the production of specific cell types, such as pancreatic β-cells.