Sanford-Burnham News Archive

Biochemist Randal J. Kaufman, Ph.D., Joins Sanford-Burnham Faculty


LA JOLLA, Calif. , July 12, 2011

Randal J. Kaufman, Ph.D., has been appointed professor and director of the Degenerative Disease Research Program at Sanford-Burnham Medical Research Institute (Sanford-Burnham) in the Institute’s Del E. Webb Neuroscience, Aging and Stem Cell Research Center.

“I am looking forward to the opportunities for collaboration that Sanford-Burnham affords,” Dr. Kaufman said. “This promises to beRandal Kaufman a very productive environment for my area of research.”

Dr. Kaufman’s current research is focused on understanding the fundamental mechanisms that regulate protein folding and the cellular responses to the accumulation of unfolded proteins within the endoplasmic reticulum (ER). When proteins fail to fold correctly, they don’t work properly. Certain types of misfolded proteins defy eradication by the cellular protein degradation machinery and accumulate with age, causing cellular toxicity. In many degenerative diseases, including neurological, metabolic, genetic and inflammatory diseases, it’s thought that the accumulation of misfolded proteins leads to cellular dysfunction and death.

“This work holds implications for the understanding of many diseases, including Alzheimer’s, Parkinson’s and Lou Gehrig’s (ALS) diseases,” said Center Director Stuart Lipton, M.D., Ph.D., who recruited Dr. Kaufman to Sanford-Burnham. “Dr. Kaufman’s arrival at Sanford-Burnham will bring new capabilities to study these and other diseases as the Institute seeks innovative new therapies to combat protein misfolding and the cellular stress it causes.”

Dr. Kaufman previously served as professor of Biological Chemistry and Internal Medicine and Howard Hughes Medical Research Institute investigator at the University of Michigan Medical School. He received his Ph.D. in pharmacology from Stanford University, where he studied gene amplification as a mechanism by which cells become resistant to anticancer agents. He was a Helen Hay Whitney fellow with Nobel Laureate Dr. Phillip Sharp at the Center for Cancer Research at the Massachusetts Institute of Technology (M.I.T.), where he developed gene transfer technologies based on gene amplification and expression in mammalian cells. He did his postdoctoral work at the Center for Cancer Research at M.I.T.

In the 1980s, Dr. Kaufman’s experience with gene transfer and engineering led him to become a founding scientist at Genetics Institute Inc., where he engineered mammalian cells for high-level expression of therapeutic proteins, such as clotting factors that are now used to treat individuals with hemophilia. His biotech industry experience makes Dr. Kaufman well-suited to Sanford-Burnham’s growing emphasis on translational medicine.

“We are excited to welcome Dr. Kaufman as part of our collaborative community of researchers here at Sanford-Burnham,” said Sanford-Burnham CEO John C. Reed, M.D., Ph.D. “As we continue to grow and attract new talent, we move ever closer to the treatments and cures that we work so hard to discover every day.”

About Sanford-Burnham Medical Research Institute

Sanford-Burnham Medical Research Institute is dedicated to discovering the fundamental molecular causes of disease and devising the innovative therapies of tomorrow. Sanford-Burnham takes a collaborative approach to medical research with major programs in cancer, neurodegeneration and stem cells, diabetes, and infectious, inflammatory, and childhood diseases. The Institute is recognized for its National Cancer Institute-designated Cancer Center and expertise in drug discovery technologies. Sanford-Burnham is a nonprofit, independent institute that employs more than 1,000 scientists and staff in San Diego (La Jolla), Calif., and Orlando (Lake Nona), Fla. For more information, visit us at

Sanford-Burnham can also be found on Facebook at and on Twitter @sanfordburnham.

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