REGULATION OF ERAD- AND UPR-RELATED GENE EXPRESSION

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. Transcript levels for various enzymes and proteins involved in human glycoprotein biosynthesis, ER-associated glycoprotein disposal (ERAD), and unfolded protein response (UPR) will be quantitated by qRT-PCR. A set of projects are being initiated in collaboration with Dr. Hung Do (Amicus Therapeutics) to examine the roles of individual genes in ER quality control and glycoprotein degradation in human cells. Amicus Therapeutics is a biotech company developing small molecule chemical chaperone therapeutics for treatment of lysosomal storage diseases. These diseases are characterized by the accumulation of misfolded lysosomal enzymes in the ER of human cells where they potentially induce an unfolded protein response.