What is the focus of our program—What questions are we asking?
Our research is focused to understand how the cell discriminates functional from nonfunctional proteins to target the latter for degradation. The answers to these questions will lead to novel therapeutics for many diseases associated with aging. We study intrinsic cellular mechanisms that recognize misfolded/damaged proteins and target their degradation. Importantly, our findings have demonstrated the damaging impact of oxidative/nitrositative stress on protein structure and function in disease pathogenesis, for example the neurodegenerative diseases of Alzheimer’s and Parkinson’s, and their impact on neural synaptic structure and signaling. Finally, defective protein folding and/or degradation are now implicated in many diseases ranging from cancer to metabolic and inflammatory response syndromes.
How will our research help patients?
Scientists in this program paved the way for development of a drug for Alzheimer’s disease that, for the first time, prevents brain cell death rather than merely masking symptoms caused by the loss of these cells. A number of other strategies are now being developed that target protein folding/degradation pathways for preserving cell function in the brain and other vital organs.