Degenerative Diseases Program - Sanford Burnham Prebys

Degenerative Diseases Program

mouse brain with alz plaque ncats

Perilous Proteins

Proteins are the workhorses of the cell. They are required for the structure, function and regulation of the body’s tissues and organs. What they do and how well they do it depends upon their three-dimensional shape, which determines whether a protein can interact with other molecules.

When proteins are misfolded or damaged, they cannot perform their duties. Faulty proteins must be eliminated before they can accumulate, clump or become toxic. Although there are intrinsic cell mechanisms that recognize defective proteins and attempt to remove them, these systems can go awry or fail as we age. Almost every age-related degenerative disease is linked to protein misfolding.


Director’s Statement

“We want to understand how cells discriminate between functional and nonfunctional proteins. We have already made important discoveries about the damaging impact of oxidative stress on protein structure and function in neurodegenerative diseases like Alzheimer’s and Parkinson’s, in metabolic diseases like diabetes and liver failure, and in inflammatory diseases and cancer. Our goal is to translate these findings into new therapies that improve or repair protein folding and preserve cell function in diseases that affect millions of people. ”

Portrait of Randal J. Kaufman, PhD
Randal J. Kaufman, PhD Program Director

Recent Publications

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