Liang Feng, PhD, Associate Professor, Molecular and Cellular Physiology, Stanford University
In this study, Dr. Liang Feng and his team want to gain a more detailed understanding of how genetic mutations involved in cystinosis affect the disease at a molecular level (e.g., how the mutations change the function or behavior of proteins or cells). They also want to investigate whether they can modify or influence important proteins that play a role in the disease, possibly to correct or reduce the harmful effects of the mutation.
In summary:
The study’s goal is twofold: (1) better understand how genetic mutations cause cystinosis at the molecular level, and (2) see if it’s possible to adjust or control certain proteins to develop new therapeutic strategies. View Research Progress Report.