University of California, San Francisco (UCSF)

Human Cell Based Model of Diabetic Neuropathy for Biomarker and Drug Discovery

Research Area

Grant Type



Despite therapeutic advances in controlling blood sugar levels, the majority of diabetic patients continue to suffer from diverse and severe long-term complications such as kidney failure, blindness and peripheral nerve damage or neuropathy. Of these, diabetic peripheral neuropathy (DPN) is the most common complication. The progression of nerve damage can ultimately lead to organ failure and amputation. Over the past decades, all tested candidate drugs have failed at different stages of clinical trials. This is largely linked to the limitations of the experimental systems in which candidate drugs were initially identified or validated, and the extent of tissue damage at the time of diagnosis. Considering the restricted repair capacity of nerves, preventative and early-stage treatment strategies are key to successful management of DPN, but unfortunately there is currently no early stage diagnostic tool available. In recent years, human pluripotent stem cell technology has provided an excellent opportunity for a better understanding of diseases in human cells. Using this technology I am developing an innovative disease-modeling framework to understand the mechanism of diabetic nerve damage, identify new candidate drugs and develop a method for accurate early diagnosis of DPN with the goal of facilitating timely interventions in future.