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Director of the Degenerative Diseases Program, Endowed Chair Cell Biology, Sanford Burnham Prebys Discovery Institute Dr. Randal J. Kaufman has made fundamental contributions to translational medicine in his industrial and academic careers. He received a Ph.D. in Pharmacology from Stanford University in 1979 where he discovered that drug resistance in cancer cells results from gene amplification. He performed post-doctoral work with Nobel laureate Dr. Phillip Sharp at the MIT Center for Cancer Research studying gene amplification and expression in mammalian cells with the goal to develop expression vectors. After post-doctoral work, he was a founding scientist at the Genetics Institute Inc., a biotechnology company where his team isolated clotting factor genes to produce recombinant proteins that are presently used throughout the world for hemophilia treatment. He also developed technologies for expression cloning of genes and high-level production of therapeutic proteins in mammalian cells. In 1994, he moved to the University of Michigan Medical Center where he became Investigator in the HHMI and Professor in the Department of Biological Chemistry and Warner-Lambert Chair in Medicine where he studied rate-limiting steps in protein folding and secretion. His studies in this arena were paradigm-shifting toward future genetic engineering of mammalian cells to efficiently secrete therapeutic proteins and contributed to the discovery of the unfolded protein response (UPR). In 2011, he moved to take position as Director and Professor in the Degenerative Diseases Program and SBP Medical Discovery Institute, La Jolla Ca. After elucidating the molecular sensors and mechanisms that signal the UPR through PERK, IRE1 and ATF6, Kaufman extended his studies using murine genetic models to show that UPR signaling is essential for normal physiology and also contributes to the progression of diverse pathologies including metabolic syndrome, diabetes, inflammation, neurodegeneration and cancer. Randal Kaufman, PhD
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