Samagya Banskota, PhD

Wolfram Syndrome is a rare genetic disorder that manifests in children as young as 6 years old. Currently, there is no cure, and patients typically succumb prematurely, with a mean lifespan of 30 years. This underscores the critical need for innovative therapeutic solutions. My proposed research endeavors to harness base editing, a precision genome editing technology, to rectify mutations in the Wfs1 gene associated with Wolfram Syndrome. By pioneering the first in vivo precision genome editing therapeutic strategy for this condition, I aim to address its underlying cause directly. This approach holds immense potential to transform patient care by offering a singular therapeutic intervention capable of effectively curing Wolfram Syndrome. Furthermore, my work will also help pave the way to advance gene editing treatments for other rare genetic disorders impacting approximately one in ten Americans, with nearly half of these cases occurring in children.