Juan J. Gallardo-Pinera, Bachelors of Science in Molecular Biology

Washington University in St. Louis (WU)

My project aims to conduct preclinical research focused on treating Wolfram Syndrome, a genetic disorder, through the innovative application of CRISPR prime editing. This approach will be utilized in vivo for gene therapy in mutant mice models that exhibit this disorder, as well as in retinal ganglion cell and brain organoids. The success of this research could expedite the progression to clinical trials, potentially paving the way for the first-ever cure for Wolfram Syndrome. This condition, known for causing neurodegeneration, blindness, deafness, diabetes, and significantly reduced lifespan (death at occurs around 30 years of age), could see a groundbreaking therapeutic advancement through this study.