GENERAL CLINICAL RESEARCH CENTER

这是一项更新多门类综合临床的建议 儿童医院和大学医院的研究中心。主要领域 研究包括： 1.儿科肝病。研究正在定义病理生理学和 新的先天性胆汁酸代谢异常和过氧化物症的治疗 疾病与骨病和生长发育障碍的发病机制 胆汁淤积性肝病。 2.骨骼健康。研究将检查产妇年龄在 哺乳对骨脱矿的影响及孕激素的应用价值 减少损失。研究将评估钙的作用 补充剂对青春期前儿童骨生长的作用 运动对学龄前儿童骨吸收的影响及其发病机制 幼年类风湿性关节炎的骨病处理。 3.癌症。使用通过建议的组织采购提供的资源 由GCRC、基础科学和临床研究人员支持的设施 将获得肿瘤用于细胞生物学和分子研究 恶性肿瘤的机制。研究将调查甲状腺癌， 神经肿瘤、乳腺癌以及APC和Bloom综合征基因。 4.高谢病。尽管酶的研究可以用来 高谢病，因骨病致残的情况持续存在。研究将会 评估双膦酸类作为酶的辅助物的潜在价值 替代治疗高谢病的骨病。 5.囊性纤维化。研究正在定义一种 复制缺陷型重组腺病毒载体的构建 鼻腔上皮细胞的跨膜电导基因。 6.胆固醇合成。研究正在评估这种药物的潜在影响 母乳或婴儿配方奶粉中的胆固醇含量和胆固醇 合成率来确定人类早期是否暴露于胆固醇 婴儿可能会对胆固醇的合成产生“印记”效应。

This is a proposal for the renewal of a multi-categorical General Clinical Research Center at the Children's and University Hospitals. Major areas of research include: 1. Pediatric Liver Disease. Studies are defining the pathophysiology and treatment of new inborn errors of bile acid metabolism and peroxisomal disorders and the pathogenesis of bone disease and growth failure in cholestatic liver disease. 2. Bone Health. Studies will examine the effect of maternal age during lactation on bone demineralization and the value of progestins in ameliorating the loss. Studies will evaluate the role of calcium supplementation on bone accretion in prepubertal children, the role of exercise in bone accretion in preschoolers and the pathogenesis and management of bone disease in juvenile rheumatoid arthritis. 3. Cancer. Using resources available through a proposed Tissue Procurement Facility supported by the GCRC, basic science and clinical investigators will obtain tumors for investigations of the cell biology and molecular mechanisms of malignancies. Studies will investigate thyroid cancer, neural tumors, breast cancer, and the APC and Bloom's Syndrome genes. 4. Gaucher Disease. Despite the availability of enzyme research for Gaucher Disease, disability from bone disease persists. Studies will evaluate the potential value of bisphosphonates as an adjunct to enzyme replacement for the treatment of bone disease in Gaucher Disease. 5. Cystic Fibrosis. Studies are defining the safety of a replication-deficient recombinant adenovirus construct to deliver the CF transmembrane conductance gene to the nasal epithelium. 6. Cholesterol Synthesis. Studies are evaluating the potential effect of the cholesterol content in breast milk or infant formulae and cholesterol synthesis rates to determine if early exposure to cholesterol in the human infant may have an "imprinting" effect on cholesterol synthesis.