Team:ASIJ Tokyo



Using CRISPR Technology to Combat Alpha-1 Antitrypsin Deficiency


Alpha-1 antitrypsin (A1AT) deficiency is a genetic disorder that arises from a single base pair mutation in the SERPINA gene. The disease results in the production of a form of A1AT prone to polymerization, which builds up in liver cells and is unable to inhibit proteases in the lungs. The lack of antitrypsin leads to damage in both the liver and the lungs. Using CRISPR-Cas9 technology, we attempted to fix the point mutation in SERPINA1 so that proper antitrypsin can be produced. We will test our system in E. Coli cells using histidine tags, osmY secretion tags, and GFP as a reporter. We hope to design a liver organ bud using iPS cell technology to deliver functional antitrypsin through collaboration with Dr. Kagimoto of Healios Japan KK.