DRUG DISCOVERY FOR GAUCHER'S DISEASE

戈谢氏病的药物发现

• 批准号: 8169354
• 负责人: Jennifer Kelley
• 金额: $ 3.35万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-05-01 至 2011-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8169354
• 关键词: Chemicals; Computer Retrieval of Information on Scientific Projects Database; Data; Disease; Drug effect disorder; Enzymes; Funding; Gaucher Disease; Grant; Institution; Molecular Chaperones; National Cancer Institute; Neuropathy; Research; Research Personnel; Resources; Source; United States National Institutes of Health; beta Glucosidases; beta-Glucosidase; drug discovery; mutant; virtual

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This project has used AutoDock in a virtual screen of the National Cancer Institute Diversity data against the Beta-glucosidase mutant enzyme that is found in people suffering Gaucher Disease, a lysosomal storage disorder causing severe neuropathy. The action of drugs on this mutant is to act as a "chemical chaperone" to assist in the proper folding of the functioning enzyme.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 该项目在国家癌症研究所多样性数据的虚拟屏幕中使用AutoDock，以对抗在戈谢病患者中发现的β-葡萄糖苷酶突变酶，戈谢病是一种导致严重神经病变的溶酶体储存疾病。 药物对这种突变体的作用是作为一种“化学伴侣”，以帮助功能酶的正确折叠。