Our gene therapy research focuses on modifying the gene mutations to eradicate the disease phenotype rather than minimizing the phenotypes by targeting molecular mechanisms. We do this by utilizing the CRISPR technique of prime editing and optimizing conditions for our in-lab models of Wolfram Syndrome, such as patient-derived induced pluripotent stem cells (iPSCs). The current goal of the Urano Lab is to identify a gene therapy method with high efficacy of modification on the desired gene segments combined with low cell death for future treatment of Wolfram Syndrome, retinal dystrophy, neurodegeneration, and diabetes.
Selected Associated publications
Gene Therapy is a new research field to the lab. Publications coming soon..