REGULATION OF ERAD- AND UPR-RELATED GENE EXPRESSION

这个子项目是许多利用资源的研究子项目之一 由NIH/NCRR资助的中心拨款提供。子项目的主要支持 而子项目的主要调查员可能是由其他来源提供的， 包括其它NIH来源。 列出的子项目总成本可能 代表子项目使用的中心基础设施的估计数量， 而不是由NCRR赠款提供给子项目或子项目工作人员的直接资金。 将通过qRT-PCR定量参与人糖蛋白生物合成、ER相关糖蛋白处置（ERAD）和未折叠蛋白反应（UPR）的各种酶和蛋白的转录水平。 与Hung Do博士（Amicus Therapeutics）合作启动了一系列项目，以研究单个基因在ER质量控制和人类细胞中糖蛋白降解中的作用。Amicus Therapeutics是一家生物技术公司，开发用于治疗溶酶体贮积病的小分子化学伴侣疗法。 这些疾病的特征在于错误折叠的溶酶体酶在人细胞的ER中的积累，其中它们潜在地诱导未折叠的蛋白质应答。

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.