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Jeffery W. Kelly | |
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The central theme of our research is to understand the mechanism(s) of protein folding and misfolding, and to decipher how these processes are related to normal physiology and disease using chemical, biophysical, and cell biological approaches. Manipulations of the kinetics and thermodynamics of protein folding and/or misfolding using carefully designed small molecules synthesized by our laboratory is leading to the discovery of first in class drugs for the amelioration of loss-of-function diseases such as Gaucher disease, and gain-of-toxic function neurodegenerative diseases such as Alzheimer's and Parkinson's disease, and the familial amyloidoses. We are also seeking small molecules that influence protein translation, protein folding, and protein degradation in collaboration with the laboratories of Dr. Balch, Dr. Morimoto, and Dr. Ron. Furthermore, we strive to understand how the amyloid fold can be used for important mammalian functions within cells without the cytotoxicity typically observed with pathologic amyloid formation.
Last Modified on 08/05/2008